Clinical decision-making can be difficult for patients with SMA due to the lack of clinical trials comparing treatment ...
India Today on MSN6d
A costly hope: The financial burden of a rare genetic disorder in IndiaThe high cost and limited availability of treatment are still major challenges for patients with a rare genetic disorder ...
Children with spinal muscular atrophy (SMA) experience benefits from receiving a disease-modifying gene therapy, but many ...
Managed Healthcare Executive provides C-suite executives at health plans and provider organizations with news, analysis, ...
ThePrint on MSN8d
New hope for rare disease patients: Rs 900-cr govt project to get high-value drugs at lower rates in worksIndia has 8.4 to 19 crore rare disease patients, according to health ministry estimates. But current beneficiaries in most ...
Scholar Rock (NASDAQ: SRRK), a late-stage biopharmaceutical company focused on advancing innovative treatments for spinal ...
NICE has rejected routine NHS funding for Roche's Evrysdi (risdiplam) for spinal muscular atrophy drug in draft guidance, a decision which doesn't come as a surprise to patient association SMA UK.
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 ...
Zacks.com on MSN17d
Biogen's Phase II/III SMA Study for Higher Spinraza Dose Meets GoalsBiogen BIIB announced positive initial data from the pivotal cohort (Part B) of its mid to late-stage study evaluating the ...
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