(RTTNews) - Genentech, a member of the Roche Group (RHHBY), announced that the U.S. Food and Drug Administration has approved a New Drug Application or NDA for an Evrysdi (risdiplam) tablet for people ...

Muscular Dystrophy Association Marks Rare Disease Day by Spotlighting Community Stories Driving Progress in Research, Care, ...

The mother of a four-year-old boy with a rare muscle disease says screening newborn babies for the condition could "save ...

A Prescription Drug User Fee Act target date of September 22, 2025 has been set for the application. The Food and Drug Administration (FDA) has accepted for Priority Review the Biologics License ...

Spinal muscular atrophy (SMA) is a genetic neuromuscular disease affecting specialized nerve cells that control voluntary muscle movement, according to the Muscular Dystrophy Association (MDA). It can ...

The singer shared an emotional update about her twins’ health on Sunday.

The U.S. Food and Drug Administration has approved Itvisma (onasemnogene abeparvovec-brve) for the treatment of spinal muscular atrophy (SMA) in patients 2 years and older with confirmed mutation in ...

Jesy Nelson of Little Mix fame has shared the news that her twin girls have been diagnosed with SMA Type 1, a genetic ...

Days after the Little Mix alum shared her and fiancé Zion Foster’s twin daughters, 7-month-old Ocean Jade and Story Monroe, were diagnosed with Spinal Muscular Atrophy Type 1 (SMA)—a genetic disorder ...