Evrysdi was studied in patients with infantile-onset (type 1 SMA), later-onset (type 2 or type 3 SMA), and in people with a diagnosis of SMA who did not show symptoms yet (pre-symptomatic SMA).

The discussions mainly focused on apitegromab for spinal muscular atrophy (SMA), including ... which involves patients with non-ambulatory type 2/3 SMA treated with Spinraza (nusinersen) or ...

From February 12, all newborn babies will be offered screening for spinal muscular atrophy (SMA), a rare progressive neuromuscular disease. SMA will be one of more than 20 other rare disorders ...

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

For columnist Alyssa Silva, advances in the development of SMA treatments have felt like glimmers of miracles.

Application is supported by results from the DEVOTE study, which suggested that two doses of Spinraza (nusinerse) 50 mg taken ...

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.

Spinraza, also known as nusinersen, is a medicine used to treat spinal muscular atrophy (SMA ... 87% of infants were Caucasian, 2% were Black, and 4% were Asian. The average age at the time ...