A new drug-free, minimally invasive intervention targets the root cause of progressive loss of neural function in spinal muscle atrophy (SMA), an inherited neuromuscular disease.

A new spinal cord implant may help people with spinal muscular atrophy (SMA) regain some muscle function, giving the ...

Three people with a muscle-destroying disease destined to worsen got a little stronger when an implanted device zapped their ...

A Boston-area biopharmaceutical firm has submitted a biologics license application to the FDA for its investigational, fully ...

The health activists argue that the government is empowered with effective law and policy tools and therefore should ...

What is Cushing's syndrome? Cushing's syndrome is a hormonal disorder. It’s caused when you have high levels of the hormone cortisol over a long time. Cushing's syndrome is fairly rare. It most often ...

A woman with SMA type 3 became pregnant while on Spinraza treatment and gave birth to a healthy child, per a case report.

Five more rare diseases have been identified in the country, making the total number of rare diseases reach 456, according to the Rare Disease Foundation.

However, recommending moderate to vigorous PA (MVPA) to patients with recent acute coronary syndrome (ACS) raises concerns owing to potential risk of recurrent ACS or fatal arrhythmias. This study ...

A combination of a ready-to-use SMN protein and an experimental treatment targeting the SMN2 gene, which works in a similar way to Spinraza (nusinersen), eased abnormalities in motor neurons derived ...