Researchers have identified a protein that’s critical to the development of skeletal muscle mass and strength and the tissue’s ability to metabolize glucose. They say their findings may lead to ...

A positive effect of nusinersen treatment on motor function in ambulant pediatric and adult spinal muscular atrophy (SMA) patients during a 38-month period has been demonstrated by an analysis of data ...

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today presented positive results from the primary analysis of the ongoing RAINBOWFISH ...

Medical researchers have uncovered a novel mechanism that leads to motor neuron degeneration in spinal muscular atrophy (SMA). This discovery offers a new target for treatment that overcomes important ...

Please provide your email address to receive an email when new articles are posted on . The study examined 26 newborns with spinal muscular atrophy given Evrysdi. Data showed 81% of babies were able ...

The Global Spinal Muscular Atrophy (SMA) Treatment Market is projected to grow at a healthy rate of around 13% by 2026. This growth is primarily driven by the increasing prevalence of SMA, growing ...

Scholar Rock (SRRK) intends to resubmit the apitegromab Biologics License Application (BLA) upon resolution of Catalent Indiana LLC-related observations CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Scholar ...

The first prenatal treatment for spinal muscular atrophy showed promise in a single case report. Risdiplam was given to the mother during pregnancy and to the child after birth. More than 2 years ...

In 2016, Spinraza became the first FDA-approved treatment for spinal muscular atrophy. Researchers have found a way the therapeutic effects of Spinraza may be improved. They discovered that pairing ...

The lab of Yongchao C. Ma, PhD, at Stanley Manne Children’s Research Institute at Ann & Robert H. Lurie Children’s Hospital of Chicago uncovered a novel mechanism that leads to motor neuron ...