Former Little Mix star Jesy Nelson revealed on social media that her twin girls have been diagnosed with a rare genetic ...

Spinal muscular atrophy (SMA) is a genetic disease that causes muscle weakness and wasting, known as atrophy. People with SMA often have difficulties moving, swallowing, sitting up, and sometimes ...

Jesy Nelson, a one-time member of the British girl group Little Mix, is opening up about the rare genetic condition her ...

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 availability of disease-modifying therapies for patients with other types of SMA suggests that these key disease characteristics in SMA 3 patients should be amenable to therapy." — Barry J. Byrne ...

Spinal muscular atrophy is a disease that is usually seen in babies and children. It makes their muscles weak and hard to move. It's a rare illness that is passed down in families. SMA isn’t the only ...

Jason Mast is a general assignment reporter at STAT focused on the science behind new medicines and the systems and people that decide whether that science ever reaches patients. You can reach Jason ...

Panelists discuss how spinal muscular atrophy is an autosomal recessive genetic disease affecting motor neurons with 3 currently approved disease-modifying therapies that restore SMN protein ...

Injections for spinal muscular atrophy (SMA) refer to two treatments that the Food and Drug Administration (FDA) has approved for SMA: nusinersen (Spinraza) and onasemnogene abeparovec-xioi (Zolgensma ...

The former Little Mix singer posted an emotional video to Instagram on Sunday, vowing that the girls will "fight all the odds ...

A multidisciplinary panel of experts discusses the clinical burden of spinal muscular atrophy on patients, families, and caregivers and considers the importance of patient education and advocacy.

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 ...