Health Canada has authorized a new drug submission for masitinib — AB Science’s add-on therapy for amyotrophic lateral sclerosis (ALS) — under the notice of compliance with conditions policy (NOC/c), designed to get new medications to patients more quickly. If granted, this would allow conditional marketing of…
The nonprofit RARE-X is creating an easily-accessible, centralized data hub for all rare disease patient data that can help researchers answer questions about existing disorders, discover new ones, and work toward finding treatments. It was spun out of the work that Nicole Boice, founder and chief engagement officer of…
In response to the National Institute of Neurological Disorders and Stroke‘s (NINDS) request for community input regarding what it should prioritize to accelerate amyotrophic lateral sclerosis (ALS) research, the ALS Association’s overarching message is this: move swiftly to help patients. NINDS, which is part of the National…
Aquinnah Pharmaceuticals has partnered with Roche to further develop its brain-penetrating, oral small molecules for the treatment of amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases. These small molecules are designed to eliminate the persistent stress granules that contribute to the buildup of toxic protein clumps in…
It’s been nearly a year since the EveryLife Foundation for Rare Diseases released its expansive report finding the total economic burden of rare disorders in the U.S. to be nearly $1 trillion.
A U.S. Food and Drug Administration (FDA) advisory committee has scheduled a virtual meeting with Amylyx Pharmaceuticals to review its application for the approval of AMX0035 to treat amyotrophic lateral sclerosis (ALS), the company announced. The online meeting to discuss data supporting the approval request is set for March 30.
The investigational antibody therapy for amyotrophic lateral sclerosis (ALS), AT-1501, will now be called tegoprubart, its maker, Eledon Pharmaceuticals, has announced. The change comes after the United States Adopted Names (USAN) Council selected tegoprubart as the unique generic, or nonproprietary, name for the therapy candidate. The company…
The ALS Canada Research Program and the Brain Canada Foundation partnered to award CA$1.125 million (about $885,600) in grants to advance the understanding, diagnosis, and treatment of amyotrophic lateral sclerosis (ALS). The nine multidisciplinary research grants, awarded late last year to teams across Canada, seek to promote global…
Tau protein levels are unusually high in the motor cortex of amyotrophic lateral sclerosis (ALS) patients with a mutation in the C9orf72 gene, a study reported. The motor cortex, a part of the cerebral cortex responsible for the planning, control, and implementation of voluntary movement, is the brain region most…
Although not thought common, laryngospasm — the sudden contraction of the vocal cords that makes speaking and breathing temporarily difficult — does affect some with amyotrophic lateral sclerosis (ALS), a study reported. Excess saliva irritating the vocal cords was the most notable trigger for laryngospasm in their work, the researchers…
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