News

The U.S. Food and Drug Administration (FDA) has published on its website a five-year action plan meant to help advance the development of — and access to — treatments that may make life better and longer for people with amyotrophic lateral sclerosis (ALS) or other rare neurodegenerative diseases. The…

NeuroSense Therapeutics’ combination therapy PrimeC led to a significant decline in certain biomarkers of amyotrophic lateral sclerosis (ALS) when used as an add-on to standard of care treatment, a study found. Standard treatment alone, meanwhile, appeared to have no impact on these disease-related biomarkers. The preliminary results come from…

Doing a muscle biopsy to look for abnormal clumps of the TDP-43 protein in the nerve fibers of muscle tissue may be useful for the early detection of amyotrophic lateral sclerosis (ALS), according to a single-center study from Japan. “It is difficult to diagnose ALS in its early stages…

High school student Cole Spector is giving people with amyotrophic lateral sclerosis (ALS) a reason to smile by becoming their pen pal. In January, Spector, who turns 17 at the end of this month, started sending cards to ALS residents in San Diego after coming up with the idea…

An at-home music therapy program aimed at improving speech, swallowing, and respiration in people with amyotrophic lateral sclerosis (ALS) was well-tolerated and feasible, according to a small pilot study. While the pilot study (NCT03604822) was not designed to test the program’s effectiveness, collected data suggest that the music…

The U.S. Food and Drug Administration has granted orphan drug designation to Tranquis Therapeutics‘ experimental therapy TQS-168 for treating amyotrophic lateral sclerosis (ALS). Orphan drug status is given to experimental therapies with promise for treating rare diseases — those affecting fewer than 200,000 people in the U.S.

TikoMed reported that ILB, its investigational therapy to protect nerve cells in amyotrophic lateral sclerosis (ALS), was well-tolerated by patients, and led to improvements in mobility and disease severity while reducing spasticity scores in a small open-label clinical trial. “These early results from this ALS-study are exciting as ILB provided beneficial…

A Phase 2b clinical trial evaluating NeuroSense Therapeutics’ experimental combination therapy PrimeC in people with amyotrophic lateral sclerosis (ALS) has enrolled its first patient. PARADIGM (NCT05357950) is the company’s next step following the completion of a Phase 2a trial (NCT04165850) wherein the therapy effectively slowed disease…

Stealth BioTherapeutics’ investigational therapy SBT-272 eased inflammation and upper motor neuron degeneration in a mouse model of amyotrophic lateral sclerosis (ALS) associated with the accumulation of toxic TDP-43 protein clumps, the company announced. Motor neurons are nerve cells that control voluntary movement and progressively die in people with ALS.

A new study is investigating the longer-term effects — over a one-year period — of the therapy candidate reldesemtiv in people with amyotrophic lateral sclerosis (ALS). Cytokinetics has launched an open-label extension (OLE) study to evaluate the safety and effectiveness of reldesemtiv over a longer course in patients…