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Results in newborns with presymptomatic SMA

Results in newborns with presymptomatic SMA

Evrysdi®️ (risdiplam) newborns living with SMA

RAINBOWFISH is an ongoing, open-label trial of Evrysdi in 26 newborns who were aged 16 to 40 days at first Evrysdi dose. These infants had a genetic diagnosis of SMA but had not yet presented with symptoms (presymptomatic SMA). Six infants who had 2 or 3 copies of SMN2 were included in an interim efficacy analysis.

Learn more about the trial

Evrysdi helped infants sit, stand, or walk

Infant sitting up
AFTER 1 YEAR
100% of infants

of infants (6/6) were able to sit, as measured by HINE-2

  • 5/6 infants could pivot/rotate
  • 1/6 infants achieved stable sit

 

Infant sitting up
AFTER 1 YEAR
100% of infants

of infants (6/6) were able to sit, as measured by HINE-2

  • 5/6 infants could pivot/rotate
  • 1/6 infants achieved stable sit

 

Infant standing up
AFTER 1 YEAR
67% of infants

of infants (4/6) were able to stand, as measured by HINE-2

  • 3/6 infants could stand unaided
  • 1/6 infants could stand with support
     
Infant standing up
AFTER 1 YEAR
67% of infants

of infants (4/6) were able to stand, as measured by HINE-2

  • 3/6 infants could stand unaided
  • 1/6 infants could stand with support
     
Infant walking independently
AFTER 1 YEAR
50% of infants

of infants (3/6) were able to walk independently, as measured by HINE-2

Infant walking independently
AFTER 1 YEAR
50% of infants

of infants (3/6) were able to walk independently, as measured by HINE-2

The Hammersmith Infant Neurological Examination–Module 2 (HINE-2) assesses 8 developmental milestones for infants, including head control, sitting, voluntary grasp, ability to kick, rolling, crawling, standing, and walking.

Evrysdi helped infants survive

Breath without permanent support
AFTER 1 YEAR
100% of infants

of infants (6/6) were alive and able to breathe without permanent support*

AFTER 1 YEAR
100% of infants

of infants (6/6) were alive and able to breathe without permanent support*

*Permanent support was defined as having a tracheostomy (a surgery where a tube is inserted in the front of the throat into the windpipe) or more than 21 days of either noninvasive ventilation support (16 or more hours a day) or being intubated (a procedure where a breathing tube is inserted down the throat and into the windpipe) to help with breathing, in the absence of an acute reversible event.

Jewelfish trial icon
Learn more about JEWELFISH, an open-label safety study including adults, children, and infants taking Evrysdi who have previously received treatment with other approved or investigational SMA medications.
Trial information
Safety
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Important Safety Information and Indication

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 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
  • 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.