Spinal muscular atrophy (SMA) is a severe neurological disease for which there is presently no cure, although current therapies can alleviate symptoms. In the search for better treatment options, ...

Cure Rare Disease (CRD) announced it has been awarded a $7.4 million grant from the California Institute for Regenerative Medicine (CIRM) to advance the development of a novel gene therapy for ...

MDA Ambassadors: Justin living with limb-girdle muscular dystrophy (LGMD) 2B, Caroline living with collagen VI muscular dystrophy, A.J. living with inclusion body myositis (IBM) and ...

A specific gene may play a key role in new treatments that prevent muscle in the body from breaking down in serious muscle diseases, muscular dystrophies. A specific gene may play a key role in new ...

The Food and Drug Administration (FDA) has approved the first gene therapy for the treatment of Duchenne muscular dystrophy (DMD) in children from age 4 through 5 years of age. Pediatric patients who ...

Neurological diseases affect the nervous system, including the brain, spinal cord, and peripheral nerves that carry signals throughout your body. These conditions can disrupt movement, thinking, mood, ...

Research Triangle Park, N.C., Nov. 07, 2024 (GLOBE NEWSWIRE) -- Asklepios BioPharmaceutical, Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, ...

Tributes flooded the internet for Gilbert Gottfried after his family shared on Tuesday via Twitter that he died following “a long illness.” Since then, there has been more awareness around muscular ...

Pompe disease is a rare genetic disorder that disables the heart and skeletal muscles. The inherited disorder can develop at any age, although the often-fatal disorder has faster progression and ...

In a new discovery, chronic pain has been shown to be physiologically different from acute pain and now scientists have the roadmap for how to target it. While many people with neuromuscular diseases ...