Clinic for Special Children.

Gordonville, Pennsylvania 17529

2 studies enrolling now · 4 studies all time

What they study most

ANM, Amish Nemaline Myopathy, Charcot-Marie-Tooth Disease With Sorbitol Dehydrogenase Deficiency (CMT-SORD), Genetic Muscle Disease, Infantile-onset Nemaline Rod Myopathy, Muscular Atrophy, Spinal, Myopathies, Nemaline, Myopathy, Myopathy, Rod, Myopathy; Hereditary, NEM5, Nemaline Myopathy 5

Studies at this site

• A Study of MZE782 in Adults With PKU — Recruiting now
• WiTNNess - TNNT1 Myopathy Natural History Study — Recruiting now
• A Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy — Completed
• Confirmatory Study of Govorestat in CMT-SORD — Withdrawn before enrolling