Neuromuscular
News
Tofersen for Treatment of Familial Amyotrophic Lateral Sclerosis
Wednesday, May 01, 2019—An early-stage trial for tofersen (BIIB067; Biogen, Cambridge, MA) an investigational drug for amyotrophic lateral sclerosis (ALS), showed that the drug is linked to slower pr…
Nusinersen Efficacy for Later-Onset Spinal Muscular Atrophy Types II and III
Wednesday, April 24, 2019—April 24, 2019 -- Children with later-onset spinal muscular atrophy (SMA) types II and III treated with nusinersen (Spinraza; Biogen, Cambridge MA) had motor function improvem…
Biomarker Study Aims to Advance Understanding of ALS Diagnosis and Treatment
Monday, April 01, 2019—A clinical study starting in late 2019 will identify and measure biomarkers in patients with amyotrophic lateral sclerosis (ALS) and evaluate the treatment effect of edaravone…
Patient Advocacy Organizations Join Forces to Improve Care for Spinal Muscular Atrophy
Thursday, March 21, 2019—Patient advocacy organizations are partnering to obtain real-world evidence to advance research, drug development, and access to therapies for children and adults with spinal …
First Cohort Fully Treated in Phase 1/2a Study of Cell Transplants for Treatment of Amyotrophic Lateral Sclerosis
Monday, March 11, 2019—In a phase 1/2a study, 5 patients with amyotrophic lateral scleroris (ALS) have had healthy astrocytes derived from stem cells (AstroRx; Kadimastem, Ness Ziona, Israel) transp…
Patient Impact Award for Liquid Riluzole Treatment for ALS
Friday, March 01, 2019—The Patient Impact Award from Life Sciences Pennsylvania pharmaceutical company will be recognized in March for the US launch of the first easy-to-swallow, thickened riluzole …
National Online Directory of Amyotrophic Lateral Sclerosis Specialists Launched
Monday, February 04, 2019—A national online database of healthcare providers (HCPs) who treat amyotrophic lateral sclerosis (ALS) has been launched by Mitsubishi Tanabe Pharma America (MTPA). The …
Neuromuscular Community Survey Identifies Key Issues
Thursday, January 17, 2019—The Muscular Dystrophy Association (MDA) has announced results of a landmark survey that asked more than 3,000 people affected by neuromuscular disease (NMD) about their key a…
