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 ...

The BBC's Ben Morris describes living with spinal muscular atrophy, as former Little Mix star Jesy Nelson reveals her twins have the condition.

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 ...

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, ...

What is SMA? The rare genetic condition that Jesy Nelson’s twins have - Former Little Mix star revealed her twin daughters had been diagnosed with Spinal Muscular Atrophy (SMA) Type 1 ...

Spinraza, also known as nusinersen, is a medicine used to treat spinal muscular atrophy (SMA) in children and adults. SMA is a genetic disease that is passed down through families. It leads to muscle ...

In his CEO Commentary, Frederic Revah, Ph.D., underscores the dedication, expertise and successes of Genethon's 240 scientists and professional staff as they pursue new gene therapy treatments for ...

Patients with SMA often suffer from impaired bulbar function, which negatively affects quality of life and can be difficult to treat effectively. A German study prospectively followed children with ...

Nelson's twin daughters, Ocean Jade and Story Monroe, have been diagnosed with spinal muscular atrophy (SMA) Type 1 ...

Evrysdi, also known as risdiplam, is an oral medicine used to treat spinal muscular atrophy (SMA) in children and adults. SMA is a genetic disease that is passed down through families. It leads to ...