Patricia Inácio, PhD,  science writer—

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…

This November, I AM ALS, a nonprofit dedicated to helping people — both patients and family members — affected by amyotrophic lateral sclerosis (ALS), is launching a campaign focused on supporting caregivers. The new initiative aims to raise awareness of the unique challenges faced by ALS caregivers. Throughout…

A database integrating long-term respiratory data with natural history data from people with amyotrophic lateral sclerosis (ALS) has been made available to scientists globally, with the hopes of accelerating research and the discovery of new therapies. Developed under a collaboration between the nonprofit Target ALS and Zephyrx, the…

A treatment candidate from Revalesio named RNS60 protected motor neurons from damage in a mouse model of amyotrophic lateral sclerosis (ALS) linked with accumulating toxic TDP-43 protein clumps. It also showed it could improve the health of mitochondria, the cells’ powerhouses that are abnormal in ALS, and reduce…

Modality.AI is teaming up with Target ALS on a clinical study of artificial intelligence (AI) as way to assess changes in speech and motor function in people with amyotrophic lateral sclerosis (ALS). The trial will harness a virtual, conversational AI system, called Tina, intended to detect even…