A European Medicines Agency panel recommended that the one-time gene therapy Itvisma be approved to treat SMA patients ages 2 ...

Novartis NVS announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) ...

The MarketWatch News Department was not involved in the creation of this content. SCHAUMBURG, Ill., March 30, 2026 /PRNewswire/ -- Cure SMA, the leading nonprofit organization supporting those ...

Panelists discuss how the needs of older patients with spinal muscular atrophy (SMA) differ from infants, focusing on independence, education, career goals, fatigue management, and quality of life ...

If approved, Itvisma (intrathecal onasemnogene abeparvovec) will be first and only gene replacement therapy for children two ...

WASHINGTON, Feb. 3, 2026 /PRNewswire/ -- Cure SMA today announced that key research and legislative priorities championed by the spinal muscular atrophy (SMA) community have been approved by Congress ...

Panelists discuss how supportive care for patients with spinal muscular atrophy (SMA) includes rehabilitation, respiratory care, psychosocial support, and multidisciplinary approaches to help patients ...

Brooklyn Nichols measures a good day by the simple things. Passing a test. Finishing her homework. Sitting down with her markers and filling a page with drawings. “Doing the things I want to do,” said ...

Novartis is telling people living with spinal muscular atrophy (SMA), a rare neuromuscular disease, to “SMAsh” their limits in a recently-launched campaign. SMA is a rare genetic neuromuscular disease ...

CONTINUE. REPORTING IN WASHINGTON. I’M JACKIE DEFUSCO. JACKIE. THANK YOU. CLOSER TO HOME, BACK IN SEPTEMBER, WE INTRODUCED YOU TO A SIX YEAR OLD UPSTATE GIRL WITH A RARE CONDITION KNOWN AS SMA OR ...

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 ...