- Conditions
- SMARD1, CMT2S
- Interventions
- Gene Therapy
- Biological
- Lead sponsor
- Megan Waldrop
- Other
- Eligibility
- 2 Months to 14 Years
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2030
- U.S. locations
- 1
- States / cities
- Columbus, Ohio
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- Conditions
- Spinal Muscular Atrophy, Charcot-Marie-Tooth Disease, Muscular Dystrophy, Spinal Muscular Atrophy With Respiratory Distress 1, Amyotrophic Lateral Sclerosis, Motor Neuron Disease, Neuromuscular Disease, Peroneal Muscular Atrophy
- Interventions
- sample collection
- Other
- Lead sponsor
- Nemours Children's Clinic
- Other
- Eligibility
- 1 Month and older
- Enrollment
- 230 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2015 – 2024
- U.S. locations
- 3
- States / cities
- Wilmington, Delaware • Jacksonville, Florida • Orlando, Florida