Clinical decision-making can be difficult for patients with SMA due to the lack of clinical trials comparing treatment ...

Risdiplam is a potential rival to Biogen’s Spinraza (nusinersen), a spinally injected antisense drug that tells the body to produce the SMN protein that is defective in patients with SMA and has ...

While Roche’s oral risdiplam has been tipped by some analysts to be a big contender in the market for spinal muscular atrophy (SMA) treatments, Novartis’ research chief Jay Bradner has a ...

Children with spinal muscular atrophy (SMA) experience benefits from receiving a disease-modifying gene therapy, but many ...

Biogen BIIB announced positive initial data from the pivotal cohort (Part B) of its mid to late-stage study evaluating the ...

Managed Healthcare Executive provides C-suite executives at health plans and provider organizations with news, analysis, ...

Advocacy groups say there's scope to make Risdiplam cheaper in India. "We have been asking the government to strike a deal with the manufacturer," says Alpana Sharma, co-founder of CureSMA India ...

Differences in SMA newborn screening practices in the U.S. could impact referral patterns or the timing of therapeutic ...

The high cost and limited availability of treatment are still major challenges for patients with a rare genetic disorder ...

Suggested remit: To appraise the clinical and cost effectiveness of nusinersen and risdiplam within their marketing authorisations for treating spinal muscular atrophy.

Scholar Rock (NASDAQ: SRRK), a late-stage biopharmaceutical company focused on advancing innovative treatments for spinal ...