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 are 16 years old or older (as this is when the transition to adult care begins).
- You have been diagnosed with a specific type of spinal muscular atrophy called 5q SMA, confirmed by a genetic test.
- You (or your legal guardian, if applicable) have signed a consent form agreeing to take part in the study.
Who may not be able to join:
- You have a different type of spinal muscular atrophy that is not the 5q type.
- Your SMA diagnosis has not been confirmed by genetic testing.
- You are under 16 years of age.
- You have not signed the consent form, or you have signed it previously but then decided to withdraw your consent.
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
Principal Investigator: Prof Chiara Marini-Bettolo, MD, PhD, Newcastle-upon-Tyne Hospitals NHS Trust
Phone: +4401912418610
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
World Health Organization (WHO) Motor Milestones; Revised Upper Limb Module (RULM); 6 Minute Walk Test (6MWT); The Hammersmith Functional Motor Scale Expanded (HFMSE); The Egen classification 2 (EK2) scale
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.