Nasdaq14d
Biogen Inc. Announces FDA and EMA Acceptance of Higher Dose Regimen for Nusinersen in Spinal Muscular Atrophy
SPINRAZA is an approved treatment for spinal muscular atrophy in over 71 countries ... Biogen Inc, Cambridge, MA. Finkle et al. Cure SMA 2024. “Final Safety and Efficacy Data From the SHINE ...
unilad28d
Controversy surrounding life-saving drug that patients only need one dose of but it costs over $2,000,000
It is used for the treatment of a condition called spinal muscular atrophy, SMA for short ... Many charities based in the US such as Sophia’s Cure, Cure SMA, Getty Owl Foundation, Fighting SMA, ...
WUNC21m
Spinal stimulation restored muscles wasted by rare genetic disorder
Three patients with spinal muscular atrophy had improved muscle strength and could walk farther after a month of daily spinal ...
The Hindu29d
Experts seek affordable access to medicines for treating Spinal Muscular Atrophy
Seeking urgent Union government intervention to overcome the lack of affordable access to medicines for treating Spinal Muscular Atrophy (SMA) and the need to allow local manufacturers to make ...
Business Wire8d
Scholar Rock Submits Biologics License Application (BLA) to the U.S. FDA for Apitegromab as a Treatment for Patients with Spinal Muscular Atrophy (SMA)
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Scholar Rock (NASDAQ: SRRK), a late-stage biopharmaceutical company focused on advancing innovative treatments for spinal muscular atrophy (SMA), cardiometabolic ...
The Hindu29d
14-year-old author with Spinal Muscular Atrophy inspires others with her resilience and determination
who are living with Spinal Muscular Atrophy (SMA) with the support of her mother, sister and teacher. Ms. Keya was felicitated by the Visakha Rasagna Vedika, a cultural organisation, at the Public ...
WebMD8d
Living Long-Term With SMA
So the outcome and the long-term outlook for our patients with SMA, particularly in the adult populations are that they are now stronger and living longer. And so as a result of that, they're able ...
contemporarypediatrics13d
FDA accepts sNDA for higher regimen nusinersen for spinal muscular atrophy
The FDA has accepted a supplemental New Drug Application for a higher dose regimen of nusinersen (Spinraza; Biogen Inc) for spinal muscular atrophy (SMA), according to a recent announcement from ...
SMA10d
Spinal cord gray matter may be imaging marker of SMA types 2, 3
Measuring the amount of gray matter in the spinal cord could help doctors track spinal muscular atrophy (SMA) over time and see how well treatments are working, according to a study that found ...
rarediseaseadvisor9d
High Prevalence of Spinal Asymmetry Identified in SMA Type 1
There appears to be a high incidence of spinal asymmetry in pediatric patients with type 1 spinal muscular atrophy (SMA) receiving nusinersen, according to a recently published study in the journal ...
Pharmaceutical Technology18d
Talditercept alfa by Biohaven for Spinal Muscular Atrophy (SMA): Likelihood of Approval
Talditercept alfa is under clinical development by Biohaven and currently in Phase III for Spinal Muscular Atrophy (SMA). According to GlobalData, Phase III drugs for Spinal Muscular Atrophy (SMA) ...