Latest News - Neuromuscular

Tuesday, October 16, 2018

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

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 (NCT01997229) and the REGAIN …

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Monday, October 15, 2018

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

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 medicine, which included s…

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Friday, October 12, 2018

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

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 the NEURO-TTR study (NCT0…

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Friday, October 12, 2018

Thymectomy May be Effective Only For Some Forms of Myasthenia Gravis

Although randomized trials demonstrate that treatment with thymectomy has benefits for patients with nonthymomatous antiacetylcholine receptor (AChR) antibody positive generalized myasthenia gravis (M…

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Wednesday, October 10, 2018

Muscular Dystrophy Association Funds $9.9 Million in Research Grants

The Muscular Dystrophy Association (MDA) has announced a new round of grant funding that includes second-generation precision medicine treatments, disease-specific research networks, analysis of large…

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Tuesday, October 09, 2018

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

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 announced. Beginning in A…

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Saturday, October 06, 2018

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

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 symptoms survived without trach…

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Wednesday, October 03, 2018

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

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 Muscle Society Congress in …

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Thursday, September 27, 2018

Neurostimulation of Spinal Cord Restores Assisted Ambulation

September 27, 2018 -- In a case study published in Nature Medicine(September 26, 2018), a combined approach of spinal cord stimulation and physical therapy has successfully enabled a man, ag…

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Wednesday, September 26, 2018

New Functional Outcome Measure for Charcot-Marie-Tooth Disease

The Charcot-Marie-Tooth Functional Outcome Measure (CMT-FOM) is a new performance-based measure designed to assess functional abilities in adults with CMT that has now been shown to be well tolerated …

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Wednesday, September 12, 2018

Cognitive Effects of Amyotrophic Lateral Sclerosis Increase as Disease Progresses

Although it is often referred to as a neurologic disease that attacks the body and not the mind, cognitive effects in amyotrophic lateral sclerosis (ALS) exist and have been reported in a study publis…

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Monday, September 10, 2018

FDA Approves of Oral Suspension of Riluzole for Patients With Amyotrophic Lateral Sclerosis

The Food and Drug Administration (FDA) has approved a new formulation of riluzole (Tiglutek; ITF Pharma, Berwyn, PA) for patients with amyotrophic lateral sclerosis (ALS). This is the first oral suspe…

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Thursday, September 06, 2018

Hereditary Neuropathy Foundation Hosting Patient-Focused Drug Development Meeting September 28th, 2018

The Hereditary Neuropathy Foundation (HNF) is holding an externally-led, patient-focused drug development meeting for Charcot-Marie-Tooth disease (CMT) and inherited neuropathies (IN) on Friday, Septe…

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Wednesday, September 05, 2018

Lower Extremity Radicular Pain Relieved With Tetrahydrocannabinol

In a small (n = 17 men), double-blind, placebo-controlled study using a counterbalanced within-subjects design, treatment of patients with chronic neuropathic pain with delta-9-tetrahydrocannibin…

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Wednesday, August 29, 2018

Nusinersen Effective Treatment for Type 1 Spinal Muscle Atrophy Even When Given at Age 7 Months and Up

Nusinersen (Spinraza; Biogen, Cambridge, MA), a novel therapy approved by the Food and Drug Administration (FDA) for treatment of patients with spinal muscle atrophy (SMA). Results of an expanded acce…

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