The U.S. Food and Drug Administration (FDA) has granted fast track designation to AMX0114, an experimental therapy being developed by Amylyx Pharmaceuticals, for the treatment of amyotrophic lateral sclerosis (ALS). Fast track status is designed to facilitate the development and review of treatments that have the potential to address…
The U.S. Food and Drug Administration (FDA) has granted fast track status to usnoflast, Zydus Lifesciences’ experimental oral inhibitor, for treating amyotrophic lateral sclerosis (ALS). The designation is meant to accelerate the development and review of new therapies that address unmet medical needs in serious or life-threatening conditions, and…
Brainstorm Cell Therapeutics is partnering with Minaris Advanced Therapies to manufacture NurOwn (debamestrocel), its investigational cell therapy for amyotrophic lateral sclerosis (ALS), for an upcoming Phase 3b clinical trial. The companies signed a letter of intent outlining the collaboration, under which Minaris, a contract development and manufacturing…
The Everylife Foundation for Rare Diseases is offering a new scholarship for students from the amyotrophic lateral sclerosis (ALS) community in the U.S. who are planning to enroll full time in an undergraduate program for the upcoming academic year. The Paula Kovarick Segalman Family Scholarship for ALS…
Treatment with the experimental therapy jacifusen was generally well tolerated and appeared to slow or even reverse disease progression in some people with amyotrophic lateral sclerosis (ALS) caused by mutations in the FUS gene. That’s according to data from 12 people who received the therapy as part of an…
Oral small molecule TPN-101 has been selected for testing in amyotrophic lateral sclerosis (ALS) as part of the HEALEY ALS platform trial. The decision to include TPN-101 in the platform trial (NCT04297683) was based on the treatment’s unique mechanism of action and final data from a Phase 2 clinical…
Antidepressants that suppress the rapid eye movement (REM) stage of sleep are associated with prolonged survival in people with amyotrophic lateral sclerosis (ALS), according to recent research. Because most muscles are paralyzed during REM, this could increase the risk of breathing issues, including possible respiratory failure, in people with…
Supporters will celebrate the 5th annual Lou Gehrig Day on June 2. The yearly event honors the legacy of the Major League Baseball (MLB) Hall of Famer whose high-profile struggle with amyotrophic lateral sclerosis (ALS) brought widespread attention to the rare neurodegenerative disease. Through awareness and fundraising initiatives,…
A study found that genetic mutations associated with amyotrophic lateral sclerosis (ALS) lead to problems with the function and transport of mitochondria, cellular structures needed for energy production, early in the disease course, before abnormal protein clumps are formed. “We show that the nerve cells, termed motor neurons, that…
The ALS Network will support a collaborative research effort, at California’s Lawrence Livermore National Laboratory, that will use electronic health records (EHRs) to identify existing medications that could slow the progression of amyotrophic lateral sclerosis (ALS). The $300,000 project is aimed at drug repurposing — finding medications already…
