Amino acid balance could play a role in SMA, as a study showed acids able to influence nervous system function were ...

Treatment with Spinraza (nusinersen) can help stabilize or improve motor function in adults with SMA, according to a new ...

Biogen enhances its portfolio and margins amidst market challenges and competition. Click here to find out why BIIB stock is ...

In return, Ionis will receive an undisclosed upfront payment from Sobi. It is also eligible to receive milestone payments and a tiered royalty on the drug's future sales.

Royalty Pharma plc (Nasdaq: RPRX) today announced that it will host an Investor Day in New York City on Thursday, September 11, 2025, starting at 8:30 a.m. ET. Royalty Pharma senior executives will ...

We recently compiled a list of the 12 Most Promising Biotech Stocks According to Wall Street Analysts. In this article, we ...

Since 2016, the FDA has approved three disease-modifying treatments for spinal muscular atrophy, with several ...

To Novartis, fresh late-stage data support the idea that its intrathecal drug, which has the same active ingredient as ...

After bringing Zolgensma to market in 2019 as the first gene therapy for spinal muscular atrophy, Novartis is back with an intrathecal formulation intended for older patients.

Delivery of onasemnogene abeparvovec into the intrathecal space was safe and effective for children with spinal muscular atrophy (SMA) aged 2 to 17 years, who had previously been shut out of receiving ...

Novartis' OAV101 IT gene therapy showed positive results in Phase 3 trials for SMA, with data supporting its potential as a treatment option.