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Phase 3 Results for Eculizumab for Treatment of Patients with Myasthenia Gravis

Tuesday, October 16, 2018—At the American Association of Neuromuscular and Electrodiagnostic Medicine (AANEM) in Washington, DC, October 10-12, results were presented from the phase 3 REGAIN trial (NCT…

AANEM Meeting Highlights Precision Medicine, Access to Care, and Technology for Neuromuscular Diseases

Monday, October 15, 2018—The American Association of Neuromuscular and Electrodiagnostic Medicine 34th Annual Meeting was held in Washington, DC October 10-12. The theme of the meeting was precision m…

Thymectomy May be Effective Only For Some Forms of Myasthenia Gravis

Friday, October 12, 2018—Although randomized trials demonstrate that treatment with thymectomy has benefits for patients with nonthymomatous antiacetylcholine receptor (AChR) antibody positive general…

Patisiran and Inotersen Continue to Show Efficacy and Safety in Open-Label and Expanded-Access Trials for Patients With Hereditary Transthyretin-Mediated Amyloidosis

Friday, October 12, 2018—Results from an open-label extension of the multi-center, randomized, double-blind phase 3 APOLLO study (NCT01960348) of patisiran (Onpattro; Alnylam, Cambridge, MA) and from …

Muscular Dystrophy Association Funds $9.9 Million in Research Grants

Wednesday, October 10, 2018—The Muscular Dystrophy Association (MDA) has announced a new round of grant funding that includes second-generation precision medicine treatments, disease-specific research ne…

First Patient Treated in Phase 1/2a Trial of Cell Therapy for Amyotrophic Lateral Sclerosis

Tuesday, October 09, 2018—The first-ever transplantation of astrocytes derived from stem cells (AstroRx; Kadimastem, Nes Ziona, Israel) into a patient with amyotrophic lateral sclerosis (ALS) has been …

Children with Spinal Muscle Atrophy Treated Presymptomatically With Nusinersen Are Sitting Independently and Walking

Saturday, October 06, 2018—Infants with spinal muscle atrophy (SMA), who were diagnosed through genetic testing and treated with nusinersen (Spinraza; Biogen, Cambridge, MA) prior to the onset of sympto…

Positive Results for Risdiplam—An Investigational Oral Agent for Spinal Muscle Atrophy

Wednesday, October 03, 2018—Results from clinical trials of risdiplam (RG7916; Genentech, South San Francisco, CA) for treatment of infants with spinal muscle atrophy (SMA) were presented at the World Mu…

 
 

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