Patricia Inácio, PhD, science writer —

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…

A short course of treatment with Revalesio‘s RNS60 as an add-on to standard amyotrophic lateral sclerosis (ALS) therapies extended the median survival of people with the progressive disease by about six months compared with a placebo. That’s according to up to six years of…

RAG-17, an investigational therapy from Ractigen Therapeutics, was safe and well tolerated across all tested doses in people with amyotrophic lateral sclerosis (ALS) carrying mutations in the SOD1 gene — cases in which the disease is known as SOD1-ALS. Those are the findings of an investigator-initiated trial (NCT05903690)…

NRG Therapeutics has received a grant from the nonprofit Target ALS to advance preclinical studies of its new class of oral small molecules targeting mitochondria, the energy producers of cells, as a potential treatment for amyotrophic lateral sclerosis (ALS). The 2024 In Vivo Target Validation grant program, an…

Targeting two particular proteins, called H1.2 and PARP1, may lessen the severity of amyotrophic lateral sclerosis (ALS) linked to mutations in the FUS gene, a study shows. Researchers found that a severe FUS mutation increased the interaction of the resulting FUS protein with both H1.2 and PARP1, which is involved in…