Shane was born with Spinal Muscular Atrophy Type 2, a progressive disease that causes muscle deterioration and weakness.

The evolution of functional assessments for spinal muscular atrophy (SMA) highlights the challenges of adapting pediatric ...

Spinal bulbar muscular atrophy (SBMA) causes a loss of motor neurons in the spinal cord and brainstem. It mainly affects facial and swallowing muscles and the muscles in the arms and legs.

This rare genetic disorder was just treated in the womb for the first time ever - Without treatment, those with the more ...

The mother took the drug daily for six weeks while the baby herself was given the drug orally, roughly one week after the ...

Spinal muscular atrophy, a progressive neurodegenerative disorder set in motion before birth, could be treated in utero, a ...

Vriddhi, a seven-month-old girl, is battling spinal muscular atrophy type 1, a rare progressive disease that will cause her muscles to weaken over time. The only hope for her survival is a gene ...

Spinal muscular atrophy (SMA) is a progressive neurodegenerative ... If not treated, SMA type 1 (SMA-1), the most common and severe form, results in progressive muscle weakness that leads to ...

The first prenatal therapy for spinal muscular atrophy (SMA) showed promising results, a case report indicated. More than 2 years after the ... the child was at risk for type 1 SMA, a severe ...

Spinraza treatment may help restore lipid balance to the cerebrospinal fluid (CSF) of children with SMA, according to a small ...