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A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy

Study on Apitegromab for infants under 2 with spinal muscular atrophy.

Recruiting
2 years and younger
All
Phase 2
**Study Overview:** This study aims to understand how a medicine called apitegromab works in very young children (less than 2 years old) who have a condition called Spinal Muscular Atrophy (SMA). SMA is a disease that makes muscles weak because of problems with genes (parts of our DNA that decide how our body works). **Apitegromab** is a medicine being tested to see if it can help improve muscle strength. **Participation Details:** - **Duration:** The study involves multiple doses, meaning you will need to visit the study location several times. - **Eligibility:** To be part of the study, children must be under 2 years old and have specific genetic markers for SMA. They also need to have been treated with certain approved SMA therapies. - **Risks/Requirements:** Children with unstable nutrition or severe bone issues may not be able to join. Regular health checks will be needed to monitor the child's progress and safety. If you are interested in learning more or considering joining the study, please contact the study team for detailed information.
Study details
    Spinal Muscular Atrophy
    SMA
    Spinal Muscular Atrophy Type 2
    Spinal Muscular Atrophy Type 3
    Neuromuscular Manifestations
    Anti-myostatin

NCT07047144

Scholar Rock, Inc.

6 June 2026

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