SMA – media

New Data Presented at MDA Clinical Conference Show Benefit in Motor Function for Infants, Teens and Young Adults Treated With SPINRAZA® (nusinersen)

Biogen (BIIB) today announced new interim Phase 2 results from NURTURE, the ongoing open-label, single-arm study evaluating the efficacy and safety of SPINRAZA® (nusinersen) among pre-symptomatic infants with spinal muscular atrophy (SMA). In NURTURE, all infants treated with SPINRAZA were alive, did not require permanent ventilation and showed improvement in motor function and motor milestone achievements as of July 5, 2017, compared to the disease’s natural history. This study, along with a case series demonstrating SPINRAZA’s effectiveness among teens and young adults, will be presented at the Muscular Dystrophy Association (MDA) Clinical Conference on March 11-14, 2018, in Arlington, Virginia.
- Source: finance.yahoo
- 2,237
- March 14, 2018
Biogen Clinical Trials SMA SPINRAZA
Scholar Rock scoops $47M to trial SMA drug

Scholar Rock has raised $47 million to take its treatment for spinal muscular atrophy (SMA) into the clinic. The series C tees up Scholar Rock to find out whether its myostatin blocker can best the underwhelming performance of one-time rivals from companies including Novartis.
- Source: fiercebiotech
- 494
- January 4, 2018
blocker Company Insight myostatin SMA
Clinical trial shows treatment is ‘transformative’ for babies with spinal muscular atrophy

Infants with the most severe form of spinal muscular atrophy (SMA) were more likely to show gains in motor function and were 47 percent more likely to survive without permanent assisted ventilation support when treated with a new medication, according to a study published today in the New England Journal of Medicine. The drug, nusinersen, performed so well that the study was stopped early and the treatment was approved shortly thereafter by the U.S. Food and Drug Administration (FDA) for all patients with this progressive neuromuscular disorder.
- Source: sciencedaily
- 453
- November 17, 2017
Market Views neuromuscular disorder SMA

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