Spinal muscular atrophy type 1 is a genetic disorder that typically appears within the first six months of life, causing severe muscle weakness and hypotonia. Babies with SMA often struggle to meet ...

19) in The New England Journal of Medicine. The child in this case specifically had SMA type 1, the most common form of the disorder; it has a very poor prognosis, typically leading to death ...

It’s a debilitating condition that affects 1 per 8,000 to 10,000 people worldwide, impacting their ability to walk, eat, and breathe. SMA Type 1 represents an estimated 50% of all cases ...

Doctors in the US have treated a fetus with a rapidly progressive congenital disease while in the uterus for the very first ...

A child with a rare genetic disorder — spinal muscular atrophy type I — was treated prenatally for the first time. Courtesy of Jakob Owens  via Unsplash During an amniocentesis test, a long, thin ...

They carried out what was essentially a 'clinical trial of one' in the patient, whose parents were both carriers of SMA type 1 and had previously had a child with the disorder – before current ...

Posters presented at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference show that therapeutic ...

Children with SMA given the gene therapy Zolgensma in infancy are maintaining motor milestones after up to 10 years, new ...