News

The National Organization for Rare Disorders (NORD) has updated its State Report Card to make it more digitally friendly and added telehealth to its categories of rare disease policy issues in a nod to its increased use during the ongoing COVID-19 pandemic. NORD’s report card project began seven…

A first healthy volunteer has been enrolled in a Phase 1 trial and given a first dose of PrimeC, an investigational combination therapy for people with amyotrophic lateral sclerosis (ALS), the treatment’s developer, NeuroSense Therapeutics, announced. The open-label Phase 1 trial (NCT05232461) is a pharmacokinetic study,…

ALPHA-0602, an investigational gene therapy for amyotrophic lateral sclerosis (ALS), can successfully increase progranulin levels and reduce the toxicity of TDP-43 protein aggregates in cell cultures and mouse models, Alpha Cognition, the therapy’s developer, announced. “These new insights from our preclinical research further support the development of ALPHA-0602…

Based on data from a Phase 1 clinical trial, Biogen and Ionis Pharmaceuticals are discontinuing development of BIIB078, their experimental treatment candidate for amyotrophic lateral sclerosis (ALS) caused by mutations in the C9orf72 gene. While the therapy was generally safe and well-tolerated in people with C9orf72-associated ALS, it…

Team Drea Foundation is launching a customizable wellness challenge that seeks to raise $50,000 in 50 days for amyotrophic lateral sclerosis (ALS) research. The 50for50 challenge invites people to set a personal goal related to the number 50, such as 50 pushups a day or 50 acts…

Minnesota lawmakers have passed legislation that will provide $25 million for amyotrophic lateral sclerosis (ALS) research and to support ALS caregivers in the state. Under the law (SF 3372), the Minnesota Office of Higher Education will receive $20 million to award research grants to scientists studying all areas of…

A CRISPR-based gene editing system could be used to reduce the activity of genes associated with amyotrophic lateral sclerosis (ALS) and Huntington’s disease, a new study shows. The study, “Targeted gene silencing in the nervous system with CRISPR-Cas13,” was published in Science Advances. CRISPR is a strategy that…

Treatment with Radicava (edaravone) for at least one year significantly reduces the risk of death in people with amyotrophic lateral sclerosis (ALS), prolonging their survival for about six months compared with those not given the therapy, an analysis of U.S. real-life data shows. “Real-world data may provide additional insight…

Synchron’s Stentrode, an innovative, implantable brain computer interface (BCI), was found safe in four amyotrophic lateral sclerosis (ALS) patients and let them communicate and perform daily online tasks by using “just their thought,” according to one-year data from a clinical trial. The results were presented as part of…

There is no substantial evidence supporting the efficacy of Amylyx Pharmaceuticals’ AMX0035 for the treatment of amyotrophic lateral sclerosis (ALS), according to a 6–4 vote by a U.S. Food and Drug Administration (FDA) advisory committee. The close vote against the therapy came at the end of a virtual…