Journal of neuromuscular diseases
Report of the multistakeholder drug development round table meeting of the World Duchenne Organization focusing on challenges for clinical development of therapies.
Annemieke Aartsma-Rus
Published: 202510.1177/22143602251399511
Abstract
Duchenne muscular dystrophy (DMD) is a muscle wasting disease where patients lose muscle tissue and function. The disease is caused by pathogenic variants that abolish the production of functional dystrophin protein. There are many therapeutic approa…
Preview only. Read the full abstract at the source