Semantic Coherence: Genentech / Evrysdi – Signal Evidence & AI Readability

[H1] Some things get everywhere
[H5] In spinal muscular atrophy (SMA) treatment, that's what you want

See how Evrysdi works

[H1] Some things get everywhere
[H5] In spinal muscular atrophy (SMA) treatment, that's what you want

See how Evrysdi works

[H2] Evrysdi is an oral, non-invasive treatment designed to produce SMN protein throughout the body.*
*This was observed when Evrysdi was studied in animals.

[IMG: Evrysdi®️ (risdiplam) navigate your SMA journey]

[H2] Navigating your SMA journey
[H4] Treatment can help preserve motor function

See how

[IMG: Here from your peers]

[H2] Hear from your peers
[H4] Connect with SMA community members who have experience with Evrysdi

Get connected

[IMG: Studied in children and adults]

[H2] Studied in adults & children
[H4] See the impact Evrysdi had in others with SMA

Explore the data

[H3] The Evrysdi community is everywhere
[H2] 18,500+
people
globally,
[H3] including people up to 75 years of age*†
*Based on individuals with SMA receiving Evrysdi worldwide as of February 2025.
†Clinical trials of Evrysdi did not include people aged 65 and older to determine whether they respond differently from those who are younger.

[IMG: World map highlighting Evrysdi®️ (risdiplam) locations globally with markers representing different countries where Evrysdi is approved]

Markers represent countries where Evrysdi is approved
As of November 2024.

[H3] Evrysdi gets here, there, EVERYWHERE
Watch our ambassadors doing the things they love.

View Transcript

Some things get everywhere
In SMA Treatment, that’s what you want.
Evrysdi is an oral, non-invasive treatment designed to reach parts of the body that need SMN protein, such as the brain and spinal cord, muscles, larynx, GI system and heart.
Evrysdi gives you more choices in how you treat SMA.
See if Evrysdi is right for you.

Evrysdi is a prescription medicine used to treat spinal muscular atrophy (SMA) in children and adults. Evrysdi works by targeting and modifying the SMN2 gene, allowing the body to produce more survival motor neuron (SMN) protein. This helps to slow down the progression of SMA and improve motor function.
Evrysdi comes in two oral, non-invasive forms, liquid and tablet. It is a daily medication that can be taken at home, at work, or on-the-go.* The recommended dosage of Evrysdi is determined by age and body weight.
*If refrigeration is not available, Evrysdi liquid can be kept at room temperature up to 104°F (40°C) for a combined total of 5 days. Please refer to the Instructions for Use for additional information about storage and administration.
The most common side effects of Evrysdi in adults and children with later-onset SMA include fever, diarrhea, and rash. The most common side effects in infants with infantile-onset SMA include fever, diarrhea, rash, runny nose, sneezing, sore throat, constipation, vomiting, and cough. These are not all the possible side effects of Evrysdi.
The safety profile of presymptomatic infants is consistent with the safety profile for symptomatic SMA patients treated with Evrysdi in clinical studies.

[H4]
Important Safety Information and Indication

Back to Top

[H5] What is Evrysdi?
Evrysdi is a prescription medicine used to treat spinal muscular atrophy (SMA) in children and adults.

Before taking Evrysdi, tell your healthcare provider about all of your medical conditions, including if you:
are pregnant or plan to become pregnant, as Evrysdi may harm your unborn baby. Ask your healthcare provider for advice before taking this medicine
are a woman who can become pregnant:
Before you start your treatment with Evrysdi, your healthcare provider may test you for pregnancy
Talk to your healthcare provider about birth control methods that may be right for you. Use birth control while on treatment and for at least 1 month after stopping Evrysdi
Pregnancy Registry. There is a pregnancy registry for women who take Evrysdi during pregnancy. The purpose of this registry is to collect information about the health of the pregnant woman and her baby. If you are pregnant or become pregnant while receiving Evrysdi, tell your healthcare provider right away. Talk to your healthcare provider about registering with the Evrysdi Pregnancy Registry. Your healthcare provider can enroll you in this registry or you can enroll by calling 1-833-760-1098 or visiting https://www.evrysdipregnancyregistry.com.
are an adult male. Evrysdi may affect a man’s ability to have children (fertility). Ask a healthcare provider for advice before taking this medicine
are breastfeeding or plan to breastfeed. It is not known if Evrysdi passes into breast milk and may harm your baby
Tell your healthcare provider about all the medicines you take
If you were prescribed Evrysdi for Oral Solution, you should receive Evrysdi from the pharmacy as a liquid. If the medicine in the bottle is a powder, do not use it. Contact your pharmacist for a replacement
Avoid getting Evrysdi on your skin or in your eyes. If Evrysdi gets on your skin, wash the area with soap and water. If Evrysdi gets in your eyes, rinse your eyes with water
The most common side effects of Evrysdi include:
For later-onset SMA:
fever
diarrhea
rash
For infantile-onset SMA:
fever
diarrhea
rash
runny nose, sneezing, and sore throat (upper respiratory infection)
lung infection (lower respiratory infection)
constipation
vomiting
cough
These are not all of the possible side effects of Evrysdi. For more information on the risk and benefits profile of Evrysdi, ask your healthcare provider or pharmacist.
You may report side effects to the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. You may also report side effects to Genentech at 1-888-835-2555.
Please see full Prescribing Information for additional Important Safety Information.

Información de prescripción de Evrysdi® (risdiplam). Genentech, Inc.

Información de prescripción de Evrysdi® (risdiplam). Genentech, Inc.

Atrophy
The medical term for getting smaller, which is generally what happens to muscles when they are not stimulated by nerve cells.

BSID-III
Stands for the Bayley Scales of Infant and Toddler Development–Third Edition, which is a tool used to assess a range of physical abilities, such as sitting, rolling, and crawling.

CI
Stands for confidence interval, which shows how much a clinical result is likely to vary.

DMT
Stands for disease-modifying treatment, which in the case of SMA, stimulates production of SMN2 protein.

FDA
Stands for Food and Drug Administration.

G-tube
Stands for gastrostomy tube, which is a tube inserted through the belly that brings nutrition directly to the stomach.

Gene
The basic unit of heredity passed from parent to child, made up of sequences of DNA (deoxyribonucleic acid). They contain information that leads to the development of physical traits such as hair color.

GI system
Stands for gastrointestinal system, also called the digestive system, which refers to the group of organs that take in food and liquids and break them down into energy the body can use.

HINE-2
Stands for Hammersmith Infant Neurological Examination–Module 2, which is a tool used to assess 8 developmental milestones for infants, including head control, sitting, voluntary grasp, ability to kick, rolling, crawling, standing, and walking.

Investigational
Term used to describe medical treatments that are currently being studied in clinical trials to determine how well they work and how safe they are to take.

Larynx
Also known as the voice box, is a hollow tube in the middle of the neck, above the windpipe and esophagus. The larynx allows you to breathe and make sounds.

MFM-32
Stands for Motor Function Measure-32 Items, which is a tool designed to capture changes in head, trunk, and limb motor movements using 32 elements in a broad range of people, including those who cannot walk. It uses 3 main categories: standing/transfer movements, upper/lower body movements, and hand/foot movements.

Molecule
The smallest particle of a substance, made up of one or more atoms.

NG-tube
Stands for nasogastric tube, which is a thin, soft tube that goes in through the nose, down the throat, and into the stomach to provide nutrition or medicine to those who cannot consume by mouth.

Non-invasive
Term used to describe medicines or medical procedures that do not require inserting anything (such as a needle) through the skin or into a body opening.

Oral
Refers to anything having to do with the mouth.

PAL
Stands for Partnership and Access Liaison.

Presymptomatic
When someone has not yet displayed any symptoms but may have underlying presence of the disease, as shown through testing or other means.

Previously treated
When someone has taken either an approved or investigational medication specifically for SMA in the past.

Progression
When the symptoms of SMA get worse over time.

Progressive disease
Any condition that gets worse over time instead of improving.

Protein
Large, complex molecules that play many important roles in the body, including making up the structure of cells and ensuring proper function of tissues and organs.

RULM
Stands for Revised Upper Limb Module, which is a tool specifically designed for people 2.5 years and older living with SMA to evaluate strength in arm movements and the ability to perform certain tasks. It includes tests, such as picking up objects, like coins/tokens, tearing paper, raising a cup to the mouth, and opening a plastic container.

Scoliosis
A sideways curve of the spine. Instead of appearing straight, the spine of a person with scoliosis will have an abnormal S or C shape that leads to their hips or shoulders appearing uneven.

Sedation
A state of calmness, relaxation, or sleepiness as a result of medications called sedatives. Sedation can be used to help you relax or fall asleep for a medical procedure.

SMA
Stands for spinal muscular atrophy, which is a genetic disease affecting the central nervous system, peripheral nervous system, and voluntary muscle movement.

SMN
Stands for survival motor neuron, which is a type of protein that your muscles need to function.

Spinal cord
A tube of tissue that runs from your brain down to your lower back and acts as an important part of your nervous system. The spinal cord carries nerve signals from your brain to the rest of your body, which allow you to feel sensations such as pressure or pain.

Type 1 SMA
SMA symptoms that are present at 6 months of age or earlier. This is sometimes called “infantile onset.” Type 1 SMA is characterized by generalized muscle weakness, a weak cry, and muscle distress, often accompanied by failure to meet developmental milestones such as sitting up unassisted.

Type 2 SMA
SMA symptoms that appear between the ages of 6 to 18 months. People with Type 2 SMA cannot stand or walk without help, but they can sit without support.

Type 3 SMA
SMA symptoms that appear after 18 months of age. People with Type 3 SMA can walk independently but may have difficulty doing so or performing other movements such as running, rising from a chair, or climbing stairs.

[H1] Designed to be different

[H2] Evrysdi helps produce SMN protein throughout the body
Evrysdi is a small molecule that is taken orally and designed to reach areas of the body that need SMN protein, such as*:

[IMG: Evrysdi® (risdiplam) helps reach areas of the body that need SMN protein—brain, spinal cord, muscles, larynx, GI system, and heart]

[H5] Designed to go everywhere

*This was observed when Evrysdi was studied in animals.

[IMG: A young girl wearing a pink dress sits amidst colorful gift wrap, holding a present. A quote reads,]

[H2] Evrysdi works daily for a consistent impact on SMN protein levels
[H3] Within 4 weeks of treatment with Evrysdi, SMN protein levels in the blood approximately doubled
Results were maintained throughout 2 years across all SMA types studied.*

Infants with Type 1 SMA†

Adults and children with Type 2 or 3 SMA†

Previously treated adults, children, and infants with Type 1, 2, or 3 SMA†

Infants with Type 1 SMA†

[IMG: Line graph titled]

Adults and children with Type 2 or 3 SMA†

[IMG: Line chart titled]

Previously treated adults, children, and infants with Type 1, 2, or 3 SMA†

[IMG: Line graph titled]

*Types 1, 2, and 3 SMA. No data available in presymptomatic SMA (under 2 months).
†Evrysdi recommended dose.

[IMG: Get to know Evrysdi® (risdiplam) brochure icon]

[H4] Dive into more information about Evrysdi

Download

[H2] How Evrysdi was studied
Studied in presymptomatic and Types 1, 2, and 3 SMA

See details

[H2] Oral SMA treatment
Evrysdi gives you the choice between 2 non-invasive forms

Learn more

[H4]
Important Safety Information and Indication

Back to Top

[H5] What is Evrysdi?
Evrysdi is a prescription medicine used to treat spinal muscular atrophy (SMA) in children and adults.

Before taking Evrysdi, tell your healthcare provider about all of your medical conditions, including if you:
are pregnant or plan to become pregnant, as Evrysdi may harm your unborn baby. Ask your healthcare provider for advice before taking this medicine
are a woman who can become pregnant:
Before you start your treatment with Evrysdi, your healthcare provider may test you for pregnancy
Talk to your healthcare provider about birth control methods that may be right for you. Use birth control while on treatment and for at least 1 month after stopping Evrysdi
Pregnancy Registry. There is a pregnancy registry for women who take Evrysdi during pregnancy. The purpose of this registry is to collect information about the health of the pregnant woman and her baby. If you are pregnant or become pregnant while receiving Evrysdi, tell your healthcare provider right away. Talk to your healthcare provider about registering with the Evrysdi Pregnancy Registry. Your healthcare provider can enroll you in this registry or you can enroll by calling 1-833-760-1098 or visiting https://www.evrysdipregnancyregistry.com.
are an adult male. Evrysdi may affect a man’s ability to have children (fertility). Ask a healthcare provider for advice before taking this medicine
are breastfeeding or plan to breastfeed. It is not known if Evrysdi passes into breast milk and may harm your baby
Tell your healthcare provider about all the medicines you take
If you were prescribed Evrysdi for Oral Solution, you should receive Evrysdi from the pharmacy as a liquid. If the medicine in the bottle is a powder, do not use it. Contact your pharmacist for a replacement
Avoid getting Evrysdi on your skin or in your eyes. If Evrysdi gets on your skin, wash the area with soap and water. If Evrysdi gets in your eyes, rinse your eyes with water
The most common side effects of Evrysdi include:
For later-onset SMA:
fever
diarrhea
rash
For infantile-onset SMA:
fever
diarrhea
rash
runny nose, sneezing, and sore throat (upper respiratory infection)
lung infection (lower respiratory infection)
constipation
vomiting
cough
These are not all of the possible side effects of Evrysdi. For more information on the risk and benefits profile of Evrysdi, ask your healthcare provider or pharmacist.
You may report side effects to the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. You may also report side effects to Genentech at 1-888-835-2555.
Please see full Prescribing Information for additional Important Safety Information.

Información de prescripción de Evrysdi® (risdiplam). Genentech, Inc.

Información de prescripción de Evrysdi® (risdiplam). Genentech, Inc.

Atrophy
The medical term for getting smaller, which is generally what happens to muscles when they are not stimulated by nerve cells.

BSID-III
Stands for the Bayley Scales of Infant and Toddler Development–Third Edition, which is a tool used to assess a range of physical abilities, such as sitting, rolling, and crawling.

CI
Stands for confidence interval, which shows how much a clinical result is likely to vary.

DMT
Stands for disease-modifying treatment, which in the case of SMA, stimulates production of SMN2 protein.

FDA
Stands for Food and Drug Administration.

G-tube
Stands for gastrostomy tube, which is a tube inserted through the belly that brings nutrition directly to the stomach.

Gene
The basic unit of heredity passed from parent to child, made up of sequences of DNA (deoxyribonucleic acid). They contain information that leads to the development of physical traits such as hair color.

GI system
Stands for gastrointestinal system, also called the digestive system, which refers to the group of organs that take in food and liquids and break them down into energy the body can use.

HINE-2
Stands for Hammersmith Infant Neurological Examination–Module 2, which is a tool used to assess 8 developmental milestones for infants, including head control, sitting, voluntary grasp, ability to kick, rolling, crawling, standing, and walking.

Investigational
Term used to describe medical treatments that are currently being studied in clinical trials to determine how well they work and how safe they are to take.

Larynx
Also known as the voice box, is a hollow tube in the middle of the neck, above the windpipe and esophagus. The larynx allows you to breathe and make sounds.

MFM-32
Stands for Motor Function Measure-32 Items, which is a tool designed to capture changes in head, trunk, and limb motor movements using 32 elements in a broad range of people, including those who cannot walk. It uses 3 main categories: standing/transfer movements, upper/lower body movements, and hand/foot movements.

Molecule
The smallest particle of a substance, made up of one or more atoms.

NG-tube
Stands for nasogastric tube, which is a thin, soft tube that goes in through the nose, down the throat, and into the stomach to provide nutrition or medicine to those who cannot consume by mouth.

Non-invasive
Term used to describe medicines or medical procedures that do not require inserting anything (such as a needle) through the skin or into a body opening.

Oral
Refers to anything having to do with the mouth.

PAL
Stands for Partnership and Access Liaison.

Presymptomatic
When someone has not yet displayed any symptoms but may have underlying presence of the disease, as shown through testing or other means.

Previously treated
When someone has taken either an approved or investigational medication specifically for SMA in the past.

Progression
When the symptoms of SMA get worse over time.

Progressive disease
Any condition that gets worse over time instead of improving.

Protein
Large, complex molecules that play many important roles in the body, including making up the structure of cells and ensuring proper function of tissues and organs.

RULM
Stands for Revised Upper Limb Module, which is a tool specifically designed for people 2.5 years and older living with SMA to evaluate strength in arm movements and the ability to perform certain tasks. It includes tests, such as picking up objects, like coins/tokens, tearing paper, raising a cup to the mouth, and opening a plastic container.

Scoliosis
A sideways curve of the spine. Instead of appearing straight, the spine of a person with scoliosis will have an abnormal S or C shape that leads to their hips or shoulders appearing uneven.

Sedation
A state of calmness, relaxation, or sleepiness as a result of medications called sedatives. Sedation can be used to help you relax or fall asleep for a medical procedure.

SMA
Stands for spinal muscular atrophy, which is a genetic disease affecting the central nervous system, peripheral nervous system, and voluntary muscle movement.

SMN
Stands for survival motor neuron, which is a type of protein that your muscles need to function.

Spinal cord
A tube of tissue that runs from your brain down to your lower back and acts as an important part of your nervous system. The spinal cord carries nerve signals from your brain to the rest of your body, which allow you to feel sensations such as pressure or pain.

Type 1 SMA
SMA symptoms that are present at 6 months of age or earlier. This is sometimes called “infantile onset.” Type 1 SMA is characterized by generalized muscle weakness, a weak cry, and muscle distress, often accompanied by failure to meet developmental milestones such as sitting up unassisted.

Type 2 SMA
SMA symptoms that appear between the ages of 6 to 18 months. People with Type 2 SMA cannot stand or walk without help, but they can sit without support.

Type 3 SMA
SMA symptoms that appear after 18 months of age. People with Type 3 SMA can walk independently but may have difficulty doing so or performing other movements such as running, rising from a chair, or climbing stairs.

[H1] SMA treatment can make a difference at any point of your journey

[H2] You can help slow the progression of spinal muscular atrophy (SMA)
[H2]

[IMG: A man in a gym setting with a basketball team in the background. Text:]

It’s important to remember that spinal muscular atrophy (SMA) is a progressive disease that slowly weakens muscles over time. While you might not notice these subtle changes on a daily basis, they can prevent you from completing everyday tasks on your own.

[IMG: A woman in a wheelchair smiles outdoors. Besides her, text discusses the slow progression of SMA and its challenges for those with Type 2 SMA. Text: "The progression of SMA can be slow. You don]

As it stands, many individuals with SMA are not receiving treatment. Whether you or someone you care for has started and stopped treatment or has yet to start treating later-onset SMA, treatment can help preserve or improve motor function and strength.
[H2] Stabilizing disease progression can have positive outcomes
[H5] Individuals receiving DMTs have reported improvements, such as:
Achieving more motor milestones
Slowing down symptom progression
Using fewer healthcare resources

[IMG: A man dining outdoors, smiling, with a quote about maintaining abilities after treatment for Type 3 SMA. “Since I’ve been on treatment, I’m still able to eat at a table, brush my teeth and shave, all like I used to. I hope to continue to maintain these abilities over time. While these wins may seem small to some, they’re major to me.” – Jose, living with Type 3 SMA]

[H5] The 2024 Cure SMA Recommendations for Treatment Considerations emphasize the importance of treating SMA as early as possible

[IMG: Get to know Evrysdi® (risdiplam) brochure icon]

[H4] A quick summary of Evrysdi

Download

[IMG: Connect resources icon]

[H4] Hear from SMA community members

Get connected

[H2] Later on-set SMA
See results in adults and children

Explore the data

[H2] Choosing a treatment
See why Evrysdi might be right for you

Learn more

[H4]
Important Safety Information and Indication

Back to Top

[H5] What is Evrysdi?
Evrysdi is a prescription medicine used to treat spinal muscular atrophy (SMA) in children and adults.

Before taking Evrysdi, tell your healthcare provider about all of your medical conditions, including if you:
are pregnant or plan to become pregnant, as Evrysdi may harm your unborn baby. Ask your healthcare provider for advice before taking this medicine
are a woman who can become pregnant:
Before you start your treatment with Evrysdi, your healthcare provider may test you for pregnancy
Talk to your healthcare provider about birth control methods that may be right for you. Use birth control while on treatment and for at least 1 month after stopping Evrysdi
Pregnancy Registry. There is a pregnancy registry for women who take Evrysdi during pregnancy. The purpose of this registry is to collect information about the health of the pregnant woman and her baby. If you are pregnant or become pregnant while receiving Evrysdi, tell your healthcare provider right away. Talk to your healthcare provider about registering with the Evrysdi Pregnancy Registry. Your healthcare provider can enroll you in this registry or you can enroll by calling 1-833-760-1098 or visiting https://www.evrysdipregnancyregistry.com.
are an adult male. Evrysdi may affect a man’s ability to have children (fertility). Ask a healthcare provider for advice before taking this medicine
are breastfeeding or plan to breastfeed. It is not known if Evrysdi passes into breast milk and may harm your baby
Tell your healthcare provider about all the medicines you take
If you were prescribed Evrysdi for Oral Solution, you should receive Evrysdi from the pharmacy as a liquid. If the medicine in the bottle is a powder, do not use it. Contact your pharmacist for a replacement
Avoid getting Evrysdi on your skin or in your eyes. If Evrysdi gets on your skin, wash the area with soap and water. If Evrysdi gets in your eyes, rinse your eyes with water
The most common side effects of Evrysdi include:
For later-onset SMA:
fever
diarrhea
rash
For infantile-onset SMA:
fever
diarrhea
rash
runny nose, sneezing, and sore throat (upper respiratory infection)
lung infection (lower respiratory infection)
constipation
vomiting
cough
These are not all of the possible side effects of Evrysdi. For more information on the risk and benefits profile of Evrysdi, ask your healthcare provider or pharmacist.
You may report side effects to the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. You may also report side effects to Genentech at 1-888-835-2555.
Please see full Prescribing Information for additional Important Safety Information.

Información de prescripción de Evrysdi® (risdiplam). Genentech, Inc.

Información de prescripción de Evrysdi® (risdiplam). Genentech, Inc.

Atrophy
The medical term for getting smaller, which is generally what happens to muscles when they are not stimulated by nerve cells.

BSID-III
Stands for the Bayley Scales of Infant and Toddler Development–Third Edition, which is a tool used to assess a range of physical abilities, such as sitting, rolling, and crawling.

CI
Stands for confidence interval, which shows how much a clinical result is likely to vary.

DMT
Stands for disease-modifying treatment, which in the case of SMA, stimulates production of SMN2 protein.

FDA
Stands for Food and Drug Administration.

G-tube
Stands for gastrostomy tube, which is a tube inserted through the belly that brings nutrition directly to the stomach.

Gene
The basic unit of heredity passed from parent to child, made up of sequences of DNA (deoxyribonucleic acid). They contain information that leads to the development of physical traits such as hair color.

GI system
Stands for gastrointestinal system, also called the digestive system, which refers to the group of organs that take in food and liquids and break them down into energy the body can use.

HINE-2
Stands for Hammersmith Infant Neurological Examination–Module 2, which is a tool used to assess 8 developmental milestones for infants, including head control, sitting, voluntary grasp, ability to kick, rolling, crawling, standing, and walking.

Investigational
Term used to describe medical treatments that are currently being studied in clinical trials to determine how well they work and how safe they are to take.

Larynx
Also known as the voice box, is a hollow tube in the middle of the neck, above the windpipe and esophagus. The larynx allows you to breathe and make sounds.

MFM-32
Stands for Motor Function Measure-32 Items, which is a tool designed to capture changes in head, trunk, and limb motor movements using 32 elements in a broad range of people, including those who cannot walk. It uses 3 main categories: standing/transfer movements, upper/lower body movements, and hand/foot movements.

Molecule
The smallest particle of a substance, made up of one or more atoms.

NG-tube
Stands for nasogastric tube, which is a thin, soft tube that goes in through the nose, down the throat, and into the stomach to provide nutrition or medicine to those who cannot consume by mouth.

Non-invasive
Term used to describe medicines or medical procedures that do not require inserting anything (such as a needle) through the skin or into a body opening.

Oral
Refers to anything having to do with the mouth.

PAL
Stands for Partnership and Access Liaison.

Presymptomatic
When someone has not yet displayed any symptoms but may have underlying presence of the disease, as shown through testing or other means.

Previously treated
When someone has taken either an approved or investigational medication specifically for SMA in the past.

Progression
When the symptoms of SMA get worse over time.

Progressive disease
Any condition that gets worse over time instead of improving.

Protein
Large, complex molecules that play many important roles in the body, including making up the structure of cells and ensuring proper function of tissues and organs.

RULM
Stands for Revised Upper Limb Module, which is a tool specifically designed for people 2.5 years and older living with SMA to evaluate strength in arm movements and the ability to perform certain tasks. It includes tests, such as picking up objects, like coins/tokens, tearing paper, raising a cup to the mouth, and opening a plastic container.

Scoliosis
A sideways curve of the spine. Instead of appearing straight, the spine of a person with scoliosis will have an abnormal S or C shape that leads to their hips or shoulders appearing uneven.

Sedation
A state of calmness, relaxation, or sleepiness as a result of medications called sedatives. Sedation can be used to help you relax or fall asleep for a medical procedure.

SMA
Stands for spinal muscular atrophy, which is a genetic disease affecting the central nervous system, peripheral nervous system, and voluntary muscle movement.

SMN
Stands for survival motor neuron, which is a type of protein that your muscles need to function.

Spinal cord
A tube of tissue that runs from your brain down to your lower back and acts as an important part of your nervous system. The spinal cord carries nerve signals from your brain to the rest of your body, which allow you to feel sensations such as pressure or pain.

Type 1 SMA
SMA symptoms that are present at 6 months of age or earlier. This is sometimes called “infantile onset.” Type 1 SMA is characterized by generalized muscle weakness, a weak cry, and muscle distress, often accompanied by failure to meet developmental milestones such as sitting up unassisted.

Type 2 SMA
SMA symptoms that appear between the ages of 6 to 18 months. People with Type 2 SMA cannot stand or walk without help, but they can sit without support.

Type 3 SMA
SMA symptoms that appear after 18 months of age. People with Type 3 SMA can walk independently but may have difficulty doing so or performing other movements such as running, rising from a chair, or climbing stairs.

[H1] MySMA SupportTM Team is here for you

[H2] MySMA Support* is a support service from Genentech that can help provide information about Evrysdi

[IMG: Support resources icon]

The following reference is located near you.
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[H4]
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[H4] The team is available to:

[IMG: Questions icon]

answer your questions about Evrysdi

[IMG: Get to know Evrysdi® (risdiplam) brochure icon]

provide information about Evrysdi, including how to take it

[IMG: Insurance coverage icon]

help explain your insurance coverage

[IMG: Financial assistance icon]

refer you to potential Evrysdi financial assistance options

[IMG: At home delivery icon]

coordinate the preparation and delivery of your medicine with the specialty pharmacy†

A guide to help you better understand potential insurance and financial support options for Evrysdi can be found here.
*Enrollment in MySMA Support through the Evrysdi Start Form is mandatory to receive assistance through the program. Participation in MySMA Support is not necessary to receive treatment with Evrysdi.
†Specialty pharmacies are not part of Genentech and maintain independence in their operations and in their role as a healthcare provider.
[H4]
Meet the PALs
Partnership and Access Liaisons (PALs) are the local, main points of contact from Genentech here to support you. Contact a PAL for answers to questions about Evrysdi (including how it works, how to take it, what results were seen in clinical trials, and important safety information), for help understanding insurance coverage and potential financial support options, and to be connected to helpful resources.

[H4]
Additional support
While a PAL will remain the main point of contact for MySMA Support, Case Managers are part of our virtual support team. A Case Manager partners closely with PALs and healthcare providers to help you understand the health insurance process and identify potential financial support options for Evrysdi.
A specialty pharmacy may also be in touch to schedule delivery of your medicine. While not part of Genentech, the specialty pharmacy is an important part of the My SMA Support team, here to prepare and ship Evrysdi to you each month and answer questions about shipping and packaging.

MySMA Support, including the PAL, does not provide medical advice and is not a substitute for your medical team.
Your healthcare provider should always be your main resource for any questions about your health and medical care.

[H5] To contact the MySMA Support team:
[H5] Call (833) 387-9734 (Monday-Friday, 9 AM to 8 PM ET)

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[H4] Request helpful tools and resources

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[H2] Financial assistance
Financial assistance options are available

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[H2] Frequently asked questions
See responses to commonly asked questions

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[H4]
Important Safety Information and Indication

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[H5] What is Evrysdi?
Evrysdi is a prescription medicine used to treat spinal muscular atrophy (SMA) in children and adults.

Before taking Evrysdi, tell your healthcare provider about all of your medical conditions, including if you:
are pregnant or plan to become pregnant, as Evrysdi may harm your unborn baby. Ask your healthcare provider for advice before taking this medicine
are a woman who can become pregnant:
Before you start your treatment with Evrysdi, your healthcare provider may test you for pregnancy
Talk to your healthcare provider about birth control methods that may be right for you. Use birth control while on treatment and for at least 1 month after stopping Evrysdi
Pregnancy Registry. There is a pregnancy registry for women who take Evrysdi during pregnancy. The purpose of this registry is to collect information about the health of the pregnant woman and her baby. If you are pregnant or become pregnant while receiving Evrysdi, tell your healthcare provider right away. Talk to your healthcare provider about registering with the Evrysdi Pregnancy Registry. Your healthcare provider can enroll you in this registry or you can enroll by calling 1-833-760-1098 or visiting https://www.evrysdipregnancyregistry.com.
are an adult male. Evrysdi may affect a man’s ability to have children (fertility). Ask a healthcare provider for advice before taking this medicine
are breastfeeding or plan to breastfeed. It is not known if Evrysdi passes into breast milk and may harm your baby
Tell your healthcare provider about all the medicines you take
If you were prescribed Evrysdi for Oral Solution, you should receive Evrysdi from the pharmacy as a liquid. If the medicine in the bottle is a powder, do not use it. Contact your pharmacist for a replacement
Avoid getting Evrysdi on your skin or in your eyes. If Evrysdi gets on your skin, wash the area with soap and water. If Evrysdi gets in your eyes, rinse your eyes with water
The most common side effects of Evrysdi include:
For later-onset SMA:
fever
diarrhea
rash
For infantile-onset SMA:
fever
diarrhea
rash
runny nose, sneezing, and sore throat (upper respiratory infection)
lung infection (lower respiratory infection)
constipation
vomiting
cough
These are not all of the possible side effects of Evrysdi. For more information on the risk and benefits profile of Evrysdi, ask your healthcare provider or pharmacist.
You may report side effects to the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. You may also report side effects to Genentech at 1-888-835-2555.
Please see full Prescribing Information for additional Important Safety Information.

Información de prescripción de Evrysdi® (risdiplam). Genentech, Inc.

Información de prescripción de Evrysdi® (risdiplam). Genentech, Inc.

Atrophy
The medical term for getting smaller, which is generally what happens to muscles when they are not stimulated by nerve cells.

BSID-III
Stands for the Bayley Scales of Infant and Toddler Development–Third Edition, which is a tool used to assess a range of physical abilities, such as sitting, rolling, and crawling.

CI
Stands for confidence interval, which shows how much a clinical result is likely to vary.

DMT
Stands for disease-modifying treatment, which in the case of SMA, stimulates production of SMN2 protein.

FDA
Stands for Food and Drug Administration.

G-tube
Stands for gastrostomy tube, which is a tube inserted through the belly that brings nutrition directly to the stomach.

Gene
The basic unit of heredity passed from parent to child, made up of sequences of DNA (deoxyribonucleic acid). They contain information that leads to the development of physical traits such as hair color.

GI system
Stands for gastrointestinal system, also called the digestive system, which refers to the group of organs that take in food and liquids and break them down into energy the body can use.

HINE-2
Stands for Hammersmith Infant Neurological Examination–Module 2, which is a tool used to assess 8 developmental milestones for infants, including head control, sitting, voluntary grasp, ability to kick, rolling, crawling, standing, and walking.

Investigational
Term used to describe medical treatments that are currently being studied in clinical trials to determine how well they work and how safe they are to take.

Larynx
Also known as the voice box, is a hollow tube in the middle of the neck, above the windpipe and esophagus. The larynx allows you to breathe and make sounds.

MFM-32
Stands for Motor Function Measure-32 Items, which is a tool designed to capture changes in head, trunk, and limb motor movements using 32 elements in a broad range of people, including those who cannot walk. It uses 3 main categories: standing/transfer movements, upper/lower body movements, and hand/foot movements.

Molecule
The smallest particle of a substance, made up of one or more atoms.

NG-tube
Stands for nasogastric tube, which is a thin, soft tube that goes in through the nose, down the throat, and into the stomach to provide nutrition or medicine to those who cannot consume by mouth.

Non-invasive
Term used to describe medicines or medical procedures that do not require inserting anything (such as a needle) through the skin or into a body opening.

Oral
Refers to anything having to do with the mouth.

PAL
Stands for Partnership and Access Liaison.

Presymptomatic
When someone has not yet displayed any symptoms but may have underlying presence of the disease, as shown through testing or other means.

Previously treated
When someone has taken either an approved or investigational medication specifically for SMA in the past.

Progression
When the symptoms of SMA get worse over time.

Progressive disease
Any condition that gets worse over time instead of improving.

Protein
Large, complex molecules that play many important roles in the body, including making up the structure of cells and ensuring proper function of tissues and organs.

RULM
Stands for Revised Upper Limb Module, which is a tool specifically designed for people 2.5 years and older living with SMA to evaluate strength in arm movements and the ability to perform certain tasks. It includes tests, such as picking up objects, like coins/tokens, tearing paper, raising a cup to the mouth, and opening a plastic container.

Scoliosis
A sideways curve of the spine. Instead of appearing straight, the spine of a person with scoliosis will have an abnormal S or C shape that leads to their hips or shoulders appearing uneven.

Sedation
A state of calmness, relaxation, or sleepiness as a result of medications called sedatives. Sedation can be used to help you relax or fall asleep for a medical procedure.

SMA
Stands for spinal muscular atrophy, which is a genetic disease affecting the central nervous system, peripheral nervous system, and voluntary muscle movement.

SMN
Stands for survival motor neuron, which is a type of protein that your muscles need to function.

Spinal cord
A tube of tissue that runs from your brain down to your lower back and acts as an important part of your nervous system. The spinal cord carries nerve signals from your brain to the rest of your body, which allow you to feel sensations such as pressure or pain.

Type 1 SMA
SMA symptoms that are present at 6 months of age or earlier. This is sometimes called “infantile onset.” Type 1 SMA is characterized by generalized muscle weakness, a weak cry, and muscle distress, often accompanied by failure to meet developmental milestones such as sitting up unassisted.

Type 2 SMA
SMA symptoms that appear between the ages of 6 to 18 months. People with Type 2 SMA cannot stand or walk without help, but they can sit without support.

Type 3 SMA
SMA symptoms that appear after 18 months of age. People with Type 3 SMA can walk independently but may have difficulty doing so or performing other movements such as running, rising from a chair, or climbing stairs.