Neuromuscular

Several clinical trials are examining the role of stem cells, inflammation, and other pathways in ALS.

The investigational drug is in development for treatment of Duchenne muscular dystrophy patients amenable to exon 51 skipping.

The approval is a first of its kind for the rare disease, and the first in a new class of drugs—small interfering ribonucleic acid (siRNA) treatments.

The once-daily 15-mg dose resulted in a significant 12-week improvement for patients with the rare condition.

Nine of 10 patients with chronic inflammatory demyelinating polyneuropathy may benefit from a regimen of pulsed corticosteroids followed by intravenous immunoglobulin.

A third-party manufacturing issue has delayed a phase I/IIa study for Sarepta Therapeutics.

A new study has found that an RNA-binding protein called TDP-43 could be a promising new biomarker for diagnosing amyotropic lateral sclerosis.

The autologous stem cell therapy is currently in a phase III trial.

A retrospective analysis of a dose-ranging trial found that those who were administered 100-mg daily riluzole, in comparison with placebo, spend a longer period of time in stage 4 of ALS.

A novel gene therapy has demonstrated impressive early results in a small sample of 3 children with Duchenne muscular dystrophy.

A grant from the National Institute of Neurological Disorders and Stroke, which allows for concurrent phase I and II trial design review, will speed up the development of novel treatments for patients with ALS and FTD.

An expanded access program has been opened to allow patients with ALS access to BHV-0223, a sublingual formulation of riluzole, all while Biohaven Pharmaceutical prepares a new drug application for the drug.

The announcement was discouraging to many, as only 2 agents for ALS have been granted FDA approval in as many decades.

The chief medical officer and chief operating officer of Brainstorm Cell Therapeutics spoke about a host of topics related to the treatment of amyotrophic lateral sclerosis.

The treatment is the first patient-infused subcutaneous formulation of immunoglobulin to gain approval for CIDP.

Although there is no cure for ALS, there are treatments available that can improve the quality of life and prolong survival.

What neuropsychiatric conditions are relatives of people with ALS at risk for? Are other genes involved besides C9orf72?

Two more novel therapies are quickly following the first approval in 2 decades for ALS.

A genetic mutation suspected to be associated with amyotrophic lateral sclerosis risk has been confirmed through a large-scale research effort.

Serum testing for NMDAR and other autoantibodies is fraught with pitfalls, and careful clinical consideration is important.