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, ...

Spinal muscular atrophy (SMA) is a leading cause of infant mortality. Most cases of the disease are caused by mutations in a gene called SMN1 that reduce the production of functional SMN protein, ...

Jesy Nelson recently announced that her twins Ocean Jade and Story Monroe Nelson-Foster, who were born prematurely in May ...

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 ...

- Taldefgrobep alfa, a myostatin-targeting biologic investigational agent, in Phase 3 development to increase muscle mass for Spinal Muscular Atrophy patients now granted Fast Track in addition to ...

Spinal muscular atrophy 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 showing no ...

At its core, SMA is caused by a deficiency in a protein called Survival Motor Neuron (SMN) . This protein is essential for ...

University of Pittsburgh School of Medicine researchers carrying out a small pilot clinical trial demonstrating that a drug-free, minimally invasive intervention targets the root cause of progressive ...