Neuromuscular

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 …

The Importance of Sleep

Logan Schneider, MD

March/April 2019—Sleep has a critical role in brain health and is an essential part of improving neurologic care for all of our patients.

Case Report: Cyclophosphamide as Rescue Agent in Myasthenic Crisis

Amanda E. Simon, BS; and Daniel L. Menkes, MD

March/April 2019—When patients are refractory to first-line treatments for myasthenic crisis, cyclophosphamide may have benefits and warrants further study.

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 …