The tiny zebrafish is helping researchers rapidly determine whether a newborn's genetic mutation is likely to cause spinal ...

The tiny zebrafish is helping researchers rapidly determine whether a newborn's genetic mutation is likely to cause spinal ...

NBS programs are fairly new in the U.S., so not much is known about variations among them or about provider practice patterns for newborns with SMA. In a survey of providers and state NBS programs, ...

A couple whose son died from a rare genetic condition are encouraging others to take part in screening which they say could ...

More than two years after the Government committed to adding SMA to newborn testing, families are still waiting ...

Spinal muscular atrophy (SMA) Type 1 is a rare but serious genetic condition that weakens muscles and can make basic activities like eating and breathing hard for babies. Early treatment—especially ...

Spinal muscular atrophy (SMA) is a debilitating genetic condition that’s usually fatal by a few years of age. But an intriguing case study might demonstrate a simple new treatment, with a child ...

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 ...

Following the first-ever treatment for spinal muscular atrophy in the womb, physicians say a 2-year-old girl shows no signs of the rare genetic disorder. Spinal muscular atrophy is a genetic condition ...

Credit: Novartis. The approval was supported by data from phase 3 STEER study, which enrolled patients with SMA who were treatment-naive and able to sit but never able to walk independently. Itvisma ...

CHESTERFIELD COUNTY, Va. (WRIC) — Spinal Muscular Atrophy (SMA) is a genetic disease that severely weakens muscles and causes developmental delays. It affects one in 10,000 American babies in the U.S.

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 ...