News

The National Organization for Rare Disorders (NORD) is seeking individuals willing to share real-life experiences with rare diseases to speak at its upcoming virtual Living Rare, Living Stronger NORD Patient and Family Forum. The interactive, patient-focused forum will be held online June 26-27. The deadline to apply for…

Nearly half of all patients in a Phase 2 trial of CNM-Au8, Clene Nanomedicine’s lead candidate for the treatment of amyotrophic lateral sclerosis (ALS), showed improvements in the health and functioning of their motor neurons, according to the interim trial data. These data are still blinded,…

Spinogenix has been awarded a research grant from the U.S. Department of Defense (DOD) to further test its lead compound as a potential treatment of amyotrophic lateral sclerosis (ALS), the company announced. “We are pleased that the DoD has recognized the potential of our novel drug candidate to…

A system using human cells to model how the brain controls muscle movement has been developed for the first time, and scientists expect it to aid in understanding neurological disorders that affect movement, including amyotrophic lateral sclerosis (ALS). The study describing their model system, “Generation of Functional…

Mitsubishi Tanabe Pharma America (MTPA) has launched an extension study to assess the long-term effects of an investigational oral formulation of edaravone (MT-1186) for the treatment of amyotrophic lateral sclerosis (ALS). “As a company, we strive to always put patients first in our efforts to understand this debilitating…

A Phase 2b/3 trial investigating the potential of SLS-005 (trehalose) to slow the progression of amyotrophic lateral sclerosis (ALS) has been added to the HEALEY ALS Platform Trial, the first such multi-regimen study created for ALS. The trial, designed by Seelos Therapeutics, the maker of…

Target ALS, a nonprofit working to advance research, has awarded a second round of grants supporting four projects that aim to discover and develop biomarkers for amyotrophic lateral sclerosis (ALS). The grants are part of The Target ALS Diagnosis Initiative, a $15 million comprehensive effort to discover…

After years of behind-the-scenes work, the ALS Association is celebrating passage of the ALS Disability Insurance Access Act of 2019, likely to end the nearly half-year wait amyotrophic lateral sclerosis (ALS) patients go through to receive Social Security Disability Insurance (SSDI) benefits. The U.S. Senate earlier this…

A single infusion of AstroRx, an off-the-shelf, investigational cell therapy for amyotrophic lateral sclerosis (ALS), safely and significantly slows disease progression in the first three months following treatment, according to updated data from an ongoing Phase 1/2 trial. These benefits waned after six months, indicating…

BrainStorm Cell Therapeutics has opened an expanded access program (EAP) in the U.S. to allow certain amyotrophic lateral sclerosis (ALS) patients to gain access to its investigational cell-based therapy NurOwn. EAPs, also known as compassionate use programs, are intended to make investigational therapies available outside of a clinical…