XLMTM (X-linked myotubular myopathy) is a serious genetic muscle condition. It is caused by changes in the MTM1 gene which stops or slows down normal muscle de…

X-Linked Myotubular Myopathy

~Estimated

~$10 – $1,125

Astellas Gene TherapiesNCT06581146

Phase 1

A Phase 1/2, Multicenter, Open-label, Dose Escalation and Expansion Clinical Study to Evaluate the Safety, Tolerability and Preliminary Efficacy of ASP2957 in Male Participants With Invasive Ventilator-dependent X-linked Myotubular Myopathy

X-linked myotubular myopathy (XLMTM) is a rare and serious condition present at birth where the muscles do not work properly. There are currently no approved t…

X-Linked Myotubular Myopathy

~Estimated

~$10 – $7,500

Astellas Gene TherapiesNCT07052929

Molecular Analysis of Neuromuscular Disease

In the Congenital Myopathy Research Program at Boston Children's Hospital and Harvard Medical School, the researchers are studying the congenital myopathies (n…

Central Core DiseaseCentronuclear MyopathyCongenital Fiber Type Disproportion+5 more

~Estimated

~$10 – $750

Boston Children's HospitalNCT00272883