Spinal Muscular Atrophy (SMA)
Spinal Muscular Atrophy (SMA) is a rare genetic disorder that affects the motor neurons in the spinal cord, leading to progressive muscle weakness and atrophy, impacting movement and mobility.
Spinal Muscular Atrophy: Risdiplam for Children
We are studying the effectiveness and safety of risdiplam in children with spinal muscular atrophy who have not improved after gene therapy. This research aims to see if risdiplam can help these patients regain function.
What diseases are being studied?
Health conditions and diseases that the clinical trial is designed to study and treat.
Who can participate in the clinical trial?
These questions help us understand your situation so we can let the trial team know whether you might be a potential match. This is not a medical evaluation and is not part of the official screening, the study doctor will make the final decision..
- Are you diagnosed with 5q-autosomal recessive SMA (spinal muscular atrophy)?
- Have you received onasemnogene abeparvovec (Zolgensma) for SMA at least 13 weeks ago?
- Do you have two copies of the SMN2 gene?
- Have you had any major illness that required hospitalization in the last month?
- Are you currently using or have you used an anti-myostatin agent?
What experimental substances are being tested?
Experimental substances that are being investigated in this clinical trial. These are not yet approved for general use.
Ro7034067
Where is the clinical trial being conducted?
Locations and medical facilities where this clinical trial is currently recruiting or conducting research. Select the locations where you would like to participate.
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Charite Universitaetsmedizin Berlin KöR
Klinik für Pädiatrie m. S. Neurologie
Berlin, Germany
Justus-Liebig-Universitaet Giessen
Department of Child Neurology
Gießen, Germany
Instytut Pomnik Centrum Zdrowia Dziecka
Instytut Pomnik - Centrum Zdrowia Dziecka
Wawer, Poland
Sponsor: F. Hoffmann-La Roche AG
Last updated: Nov 26, 2025
Disclaimer: Parts of this content have been automatically extracted from the EU Clinical Trials registry. While we strive for accuracy, please always contact the trial site or sponsor directly for correct and official information before making any decisions about participation. View on EU Clinical Trials.