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…
The Muscular Dystrophy Association (MDA) and the International Association of Fire Fighters (IAFF) are teaming up once again, launching more than 420 “Fill the Boot” events this year to raise funds for research and care for people with neuromuscular diseases, including amyotrophic lateral sclerosis (ALS). The fundraising initiative will…
Athira Pharma has completed enrollment of the first group of healthy volunteers who’ll receive a single dose of ATH-1105, its experimental oral therapy for amyotrophic lateral sclerosis (ALS), in a Phase 1 clinical trial. The Phase 1 trial (NCT 06432647),…
Patient enrollment is now complete in an arm of the HEALEY ALS platform trial that’s testing Denali Therapeutics‘ DNL343 for the treatment of adults with amyotrophic lateral sclerosis (ALS). The HEALEY platform trial (NCT04297683), led by the Sean M. Healey & AMG Center…
