After working for months behind the scenes, the ALS Association is calling a strengthened Accelerating Access to Critical Therapies for ALS Act (ACT) a key step toward swifter development of new therapies. The revised bill (HR 8662/S. 4867) calls for $100 million over five years for amyotrophic…
A newly identified type of immune cell — a kind of immature neurophil or white blood cell — was found to be responsible for the regeneration and survival of eye and spinal cord nerve fibers that is known to be possible in mice with induced nerve injury,…
A photo of a bespectacled young boy, his red baseball cap slightly askew as he enjoys time outside, will be featured on the front cover of an upcoming calendar in the “Same But Different” contest to raise awareness about rare disorders. “A Lovely Day Out in Kew Gardens,”…
Eli Lilly and Company has acquired Disarm Therapeutics and its emerging therapies designed to prevent axonal degeneration in neurodegenerative disorders, including amyotrophic lateral sclerosis (ALS), multiple sclerosis, and peripheral neuropathy. Disarm’s new class of disease-modifying treatments, called SARM1 inhibitors, target the central driver of axonal…
Bristol Myers Squibb has entered a five-year research collaboration with the machine learning startup insitro to discover and develop new therapies for amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). Machine learning is a form of artificial intelligence that uses algorithms to analyze large amounts of data, learn…
Toxic clumps due to the buildup of the TDP-43 protein can trigger the neuronal inflammation that precedes symptoms of amyotrophic lateral sclerosis (ALS) by activating an inflammatory signaling pathway called cGAS-STING, a study reported. Its findings suggest that targeting this inflammatory signaling may be an effective way of treating ALS.
A Phase 1/2 clinical trial investigating ION541, Ionis Pharmaceuticals‘ third antisense treatment and its first for sporadic, rather than familial, amyotrophic lateral sclerosis (ALS), has started dosing, the company announced. This ascending dose safety trial (NCT04494256) is recruiting up to 70 patients…
People with diabetes mellitus, particularly those with type 2 diabetes, appear to be at a significantly lower risk of amyotrophic lateral sclerosis (ALS) than those without this metabolic disorder, a review study reported. Its findings support previous research suggesting diabetes to be a protective factor against ALS, potentially…
Catalent Biologics has agreed to manufacture NurOwn, the cell-based therapy by BrainStorm Cell Therapeutics being evaluated in a soon-to-conclude pivotal trial as a possible treatment of amyotrophic lateral sclerosis (ALS). With this agreement, Catalent will produce NurOwn under current Good Manufacturing Practices — standards set to ensure that batches…
A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like the option of continuing its use in future appointments, a series of surveys found. The surveys were conducted by the National Organization for Rare Disorders (NORD) and involved more than 800…
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