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Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA)

Ongoing study of Risdiplam for those with Spinal Muscular Atrophy.

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Phase 4

This study involves people with a condition called **Spinal Muscular Atrophy (SMA)**, a disease that weakens muscles. The study will look at how safe and effective a medicine called **risdiplam** is over a long time. Risdiplam is a medicine approved by the U.S. FDA (Food and Drug Administration) to help with SMA. The study follows participants for a long time, until they choose to leave, are lost to follow-up, or pass away. Even if participants stop taking risdiplam, they may continue in the study for follow-up checks. There’s also an optional sub-study using smartphones and sensors to check movements and swallowing.

To join, you must have a clinical diagnosis of SMA and be on risdiplam based on doctor's judgment. For the sub-study, participants must be at least 10 years old, comfortable using smartphones, and able to complete specific tests. Participants who are allergic to risdiplam or have certain mental health issues cannot join.

  • The study will last as long as participants are willing and able.
  • Participants may need to visit the clinic for assessments.
  • There might be some risks, especially for those with specific health conditions.
Study details
    Spinal Muscular Atrophy

NCT05232929

Genentech, Inc.

9 February 2025

Step 1 Get in touch with the nearest study center
What happens next?
  • You can expect the study team to contact you via email or phone in the next few days.
  • Sign up as volunteer  to help accelerate the development of new treatments and to get notified about similar trials.

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Learn more about clinical trials

What is a clinical trial?

A clinical trial is a study designed to test specific interventions or treatments' effectiveness and safety, paving the way for new, innovative healthcare solutions.

Why should I take part in a clinical trial?

Participating in a clinical trial provides early access to potentially effective treatments and directly contributes to the healthcare advancements that benefit us all.

How long does a clinical trial take place?

The duration of clinical trials varies. Some trials last weeks, some years, depending on the phase and intention of the trial.

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Compensation varies per trial. Some offer payment or reimbursement for time and travel, while others may not.

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Clinical trials follow strict ethical guidelines and protocols to safeguard participants' health. They are closely monitored and safety reviewed regularly.
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