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, ...

Imagine a child born in a family with a rare disease, whose treatment would cost more than 17 crore for a single dose. The ...

WILLIAMSPORT, Pa. — Carlee Weber struggles with everyday, normal tasks. She has been using a wheelchair since she was four years old after being diagnosed with Spinal Muscular Atrophy at the age of ...

Results from a single-center, observational, prospective study suggested that inflammatory markers could predict disease severity and treatment efficacy for patients with spinal muscular atrophy (SMA) ...

Boosting communication between the spinal nerves and the muscles using the spinal cord stimulation reverses spinal muscle atrophy (SMA) progression and could be applied to other motoneuron diseases, ...

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today that the U.S. Food and Drug Administration (FDA) has approved a New ...

REDONDO BEACH, Calif. -- Ceri and Rory Devine welcomed their first child, Rhys, into the world three months before COVID-19 shut down the country. It wasn't long before the new parents from Redondo ...

New research suggests previous assumptions about the pathogenesis of spinal muscular atrophy (SMA) are inadequate. Patients with severe infantile spinal muscular atrophy (SMA) have significant loss of ...

FRESNO, Calif. -- Richard and Kaylee Lazaro had just welcomed twins Rykker and Bekkem, but Rykker was rushed to the NICU and later diagnosed with Spinal Muscular Atrophy (SMA). He was able to receive ...

Gene therapy is linked to better nonmotor outcomes in spinal muscular atrophy type 1 compared to first-line nusinersen, new ...

Dublin, March 21, 2018 (GLOBE NEWSWIRE) -- The "Global Neurodegenerative Diseases Drugs Market to 2023 - MS Set to Dominate with Tecfidera and Ocrevus, as Disease-Modifying Pipeline Therapies for ...