Topline results from a phase 3 clinical trial showed an investigational gene therapy for young people with spinal muscular atrophy type 2 increased mobility and slowed disease progression ...
The Muscular Dystrophy Association (MDA) has selected Lily Sander from Charlotte, North Carolina, as its 2025 MDA National Ambassador. Sander joins MDA National Ambassador Ira Walker from Fort ...
Dysferlin-deficient muscular dystrophy is a devastating and untreatable disease. Using Cas9, the authors restored dysferlin in muscle stem cells from patients ex vivo and show proof-of-concept for ...
Expert diagnostic services and treatment are provided for patients with common and rare disorders of the muscular system, including muscular dystrophies. Myopathic (myo = muscle, pathy = disease) ...
Muscular dystrophy (MD) represents a group of inherited muscle disorders that primarily cause muscle weakness and muscle wasting, though some types of the disease also present with other ...
Most of the muscular dystrophies are a form of inherited disease called X-linked disorders or genetic diseases that mothers can transmit to their sons even though the mothers themselves are ...
Weak Voluntary Muscles: Skeletal Muscle Diseases, Neuromuscular Disorders, and Other Causes Neuromuscular disorders, also called skeletal muscle disorders, are conditions that affect the nerves that ...