Ionis Pharmaceuticals recently celebrated the 10th anniversary of its Surf Away+ program, a day of adaptive sports and beach fun that has welcomed people with amyotrophic lateral sclerosis (ALS) alongside those with other neurological diseases. Held Sept. 12 at the beach in Oceanside, California, the event brought together…
The ALS Association has announced the winners of its first-ever Hoffman ALS Clinic Development and Capacity Awards, two new grant programs aimed at improving access to multidisciplinary care for people with amyotrophic lateral sclerosis (ALS) across the U.S. The awards are supported by a $58 million donation from the…
The ongoing program that is providing amyotrophic lateral sclerosis (ALS) patients with access to Medicinova’s investigational therapy MN‑166 (ibudilast) outside of a clinical trial is adding new sites. The expanded access program (EAP) will enroll about 200 patients at several locations in the U.S. who are not eligible…
The U.S. Food and Drug Administration (FDA) has granted orphan drug status to Klotho Neurosciences’ gene therapy candidate for amyotrophic lateral sclerosis (ALS). The designation is intended to boost the development of potential treatments for rare diseases, or those that impact fewer than 200,000 people in the U.S. It…
Voyager Therapeutics will reassess its gene therapy program for amyotrophic lateral sclerosis (ALS) associated with mutations in the SOD1 gene after its original candidate, VY9323, was found to have toxic effects in nonhuman primate studies. The company believes the problem lies with the therapy’s payload and not the viral carrier…
The U.S. Food and Drug Administration (FDA) has lifted the clinical hold on a Phase 1 clinical trial that’s testing AMX0114, Amylyx Pharmaceuticals’ investigational treatment for amyotrophic lateral sclerosis (ALS). The hold came in November after Amylyx asked to initiate the first-in-human studies for AMX0114 at a starting…
A Phase 1 clinical trial testing RAG-17, an investigational therapy for people with amyotrophic lateral sclerosis (ALS) carrying mutations in the SOD1 gene, has dosed its first participant. The trial (NCT06556394) aims to determine the safety and tolerability, pharmacological properties, and preliminary signs of efficacy of multiple dose levels of…
Dazucorilant, an oral cortisol modulator being developed by Corcept Therapeutics, didn’t significantly slow disease progression in people with amyotrophic lateral sclerosis (ALS) over a placebo, meaning it failed to meet the primary goal of the DAZALS Phase 2 clinical trial. The study (NCT05407324), which tested dazucorilant doses…
RAG-21, a therapy Ractigen Therapeutics is developing for amyotrophic lateral sclerosis (ALS) linked to mutations in the FUS gene, has been awarded orphan drug status by the U.S. Food and Drug Administration (FDA). Orphan drug status in the U.S. is granted to products aiming to prevent, diagnose, or treat…
The European Medicines Agency (EMA) has recommended that monepantel, now named NUZ-001, be granted orphan medicinal product designation to treat amyotrophic lateral sclerosis (ALS). The positive opinion will be reviewed by the European Commission, which is expected to issue a final decision in December. If granted, the designation will provide…
