Neuromuscular

The Clinical Director of the NHGRI spoke about the impact of the NIH program and its future development.

This guide includes everything you need to know about subcutaneous immunoglobulin (Hizentra, CSL Behring) in the treatment of chronic inflammatory demyelinating polyneuropathy as well as primary immunodeficiency.

The position statement’s author noted that a lack of specificity in laws and inconsistencies in protocols has led to confusion surrounding brain death in several high-profile cases.

The program’s director spoke about its genesis and evolution into a more widespread initiative which has helped improve next-generation genome sequencing.

A proof-of-concept trial’s findings have shown that more clinical research into the potential neuroprotective effect of cannabinoids in slowing disease progression in motor neuron disease is warranted.

Gene therapy has generated excitement as a treatment or even a potential cure for inherited diseases. Among them: Duchenne muscular dystrophy.

The treatment, previously known as retigabine, was assessed in a phase 2 trial in more than 60 patients with amyotrophic lateral sclerosis.

Neurology News Network for the week of December 15, 2018.

The chief medical officer of Wave Life Sciences provided insight into the investigational DMD treatment.

The therapy met both primary and secondary end points across a broad spectrum of patients with generalized myasthenia gravis, including significant reductions in both QMG and MG-ADL scores.

WVE-210201 was granted both Orphan Drug and Rare Pediatric Disease designations from the FDA, as well as Orphan Drug status in the European Commission.

Neurology News Network for the week of December 1, 2018.

Regulatory action is expected to be made by May 2019. The therapy previously received a Breakthrough Therapy designation from the FDA.

Part 2 of the phase 2 trial in CMT is ongoing, expected to enroll 40 patients with the condition.

If approved, BHV-0223 would become the only formulation of riluzole that doesn't require swallowing tablets or liquids, offering an important delivery alternative for the standard-of-care treatment of ALS.

Therapies designed to treat neurologic conditions have made up 25% of the submissions to the FDA for a Regenerative Medicine Advanced Therapy designation.

The Carolyn and Matthew Bucksbaum Professor of Clinical Ethics at the University of Chicago spoke about the ethical considerations of spinal muscular atrophy and its treatment.

The product was being marketed for conditions including Alzheimer disease, fibromyalgia, spinal cord injury, multiple sclerosis, muscular dystrophy, Parkinson disease, and peripheral neuropathy, among others.

Neurology News Network for the week of November 10, 2018.

In its second phase 2 trial, the therapy, also known as AMO-02, resulted in improvements in cognitive function, fatigue, and neuromuscular symptoms.

Darryl De Vivo, MD, emphasized that while there's been a tremendous advance in the field of SMA, it’s important to continue tinkering around the margins to make the effective treatment increasingly better.

The Sidney Carter Professor of Neurology at Columbia University Medical Center spoke about the 127 year history of spinal muscular atrophy and how basic science has led to exciting developments.

Since the inception of the idea more than 3 decades ago and its initial development 20 years later, Sarepta Therapeutics’ micro-dystrophin gene therapy has now made its way to human trials.

The gene therapy is also being assessed for long-term safety data, as well as an additional delivery method.

A novel mesenchymal stem cell therapy produced intriguing phase II data and has moved into a randomized, pivotal phase III clinical trial.