Bionews Insights is launching a survey, in collaboration with ALS News Today, with the goal of understanding how different aspects of this disease affect an individual’s quality of life. The survey, which is expected to take about 15 minutes to complete, opens with WHOQOL-100, a set of questions developed by…
A new U.S. initiative called Rare Disease Cures Accelerator–Data and Analytics Platform — dubbed RDCA–DAP — aims to accelerate treatment innovation across rare diseases by sharing existing patient data and promoting the standardization of new data collection. Launched during a virtual workshop in September, the U.S. Food and Drug…
Clene, and its wholly owned subsidiary Clene Nanomedicine, have launched an expanded access program (EAP) in the U.S. to allow certain people with amyotrophic lateral sclerosis (ALS) to gain access to its experimental oral therapy CNM-Au8. Called CNMAu8.EAP02, the program will focus on patients who are not eligible…
Seelos Therapeutics’ experimental therapy SLS-005 (trehalose) will serve as the fifth arm of the multi-regimen HEALEY trial for amyotrophic lateral sclerosis (ALS), after being cleared by the U.S. Food and Drug Administration and the Mass General Brigham Institutional Review Board. With this final regulatory authorization, Seelos may still…
PathMaker Neurosystems was awarded $371,000 from the National Institutes of Health (NIH) to advance its neuromodulation technology as a potential new, non-invasive treatment for amyotrophic lateral sclerosis (ALS). The Small Business Technology Transfer grant, from the NIH’s National Institute of Neurological Disorders and Stroke, will enable PathMaker to…
The ALS Association is applauding the recent change of heart by the U.S. Food and Drug Administration (FDA) to consider AMX0035’s approval for amyotrophic lateral sclerosis (ALS) without requiring results from an additional clinical trial. Earlier this year, the agency had requested data from a placebo-controlled Phase 3 trial,…
Football and science seem to be disparate fields of play at first glance, but the nonprofit Uplifting Athletes is finding common ground by leveraging the popularity of college gridiron games to fund research for rare diseases. Its nearly two dozen chapters — representing college football teams across the nation…
The United Kingdom-based Motor Neurone Disease (MND) Association was commended recently at the Third Sector Awards for its efforts in the MND community. The nonprofit organization won high commendations in the “Charity of the Year: Over £5M” category at the annual awards, which recognizes the achievements of charitable organizations and…
A French national agency has cleared AB Science to resume patient enrollment in the Phase 3 clinical trial investigating masitinib in people with amyotrophic lateral sclerosis (ALS), the company announced in a press release. AB had voluntarily suspended recruitment and treatment in all its masitinib clinical studies in…
QurAlis is advancing the development of QRL-201, its “groundbreaking” lead treatment candidate for amyotrophic lateral sclerosis (ALS), and expects to initiate its first clinical trial in late 2022. The stem cell technology company said it is now conducting formal preclinical studies to assess QRL-201’s therapeutic potential, and expects…
