Spinal Muscular Atrophy Trial, Recruiting
Who may be able to join
AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI
Who might be able to join this trial:
- You have a rare disease (one that affects fewer than 200,000 people in the United States), whether it has been confirmed by a doctor or is only suspected. Examples include ALS, cystic fibrosis, sickle cell disease, Huntington's disease, Duchenne muscular dystrophy, early-onset Alzheimer's, Ehlers-Danlos syndrome, and several others (confirm with trial site for the full list).
- You, or a legally authorized person acting on your behalf, are willing and able to give formal consent to participate in the study.
- If you are a minor, you may also need to give your own agreement (called "assent") in addition to a parent or guardian giving consent.
- Participation may be possible even after a person has passed away, if a legally authorized representative gives consent on their behalf, in line with U.S. federal and state laws.
Who may not be able to join:
- You, or a legally authorized person acting on your behalf, are not able to give formal consent to participate.
- You live outside of the United States and cannot provide access to your medical records.
Important: Always verify eligibility with the trial site directly before applying.
Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.
Contact this trial
Phone: 707-641-4475
Contact details sourced from ClinicalTrials.gov. Verify directly with the trial site before attending.
GP referral letter
Print a one-page summary to share with your doctor.
Trial details
Where this trial is recruiting
Primary endpoints
Overall Survival (OS); Safety/tolerability of medications; Changes in normal development milestones; Changes in functional status; Changes in motor function; Changes in symptoms or clinical status
Can't join this trial?
Data last synced from ClinicalTrials.gov: 28 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.