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 (or your child) must agree to take part by signing a consent form — if the participant is a child, a parent or legal guardian can sign on their behalf
- You (or your child) must have a confirmed diagnosis of Spinal Muscular Atrophy (SMA, type 1–4), proven by a genetic test — this includes both people who already have symptoms and those who do not yet show symptoms
- You (or your child) must be between 0 and 21 years old on the day participation begins
- You (or your child) must already be receiving an approved medication treatment for SMA at the time of joining the trial
Who may not be able to join:
- People who need a breathing machine connected through a tube inserted into the throat or airway (invasive ventilation) due to heart or breathing problems
- People who have severe bone weakness (advanced osteoporosis) and have had multiple bone fractures before starting treatment
- People whose physical abilities have been getting worse during rehabilitation, as measured by specific movement and development tests suited to their type of SMA (confirm with trial site)
- People who are unable to cooperate or work together with a therapist during sessions
- People who have other physical reasons that would prevent them from doing exercise (confirm with trial site)
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: Marek Jóźwiak, Wiktor Dega University Orthopedic and Rehabilitation Hospital
Phone: 696052475
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
Clinical assessment of patients using Children's Hospital Of Philadelphia Infant Test Of Neuromuscular Disorders, Revised Upper Limb Module, Hammersmith Functional Motor Scale; Clinical assessment of patients using Balance test minimum 1 min., Self-walk test of at least 2 meters, 6-minute gait test, Up&Go test, 10 meters gait test, hip range of motion and muscle strength evaluation.; Functional assessment of patients using GMFM (Gross Motor Function Measure) scale; X-ray or ultrasound scan imaging assessment of the spine, hip joints and bone mineral density; Assessment of the quality of life o...
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.