Answer ALS and Cedars-Sinai have made openly available their amyotrophic lateral sclerosis (ALS) patient-based stem cell and biodata repository to researchers worldwide. The largest of its kind, the repository contains biological and clinical data from nearly 1,000 ALS patients, as well as specialized nerve cells called induced pluripotent…
Can it be that we were cheering on athletes at the Olympics only a few months ago? Where did the summer go? Looking back, I realize I spent most of my summer and early fall months distracted by the hoopla of world and national events, and now I’m exhausted. I’m…
A newly awarded grant from the ALS Association will support Asha Therapeutics‘ work to advance ASHA-624, a small molecule being developed for amyotrophic lateral sclerosis (ALS), into a first-in-human clinical trial that’s expected to begin early next year. The grant was through the Lawrence and Isabel Barnett…
The European Medicines Agency (EMA) has recommended that monepantel, now named NUZ-001, be granted orphan medicinal product designation to treat amyotrophic lateral sclerosis (ALS). The positive opinion will be reviewed by the European Commission, which is expected to issue a final decision in December. If granted, the designation will provide…
A Phase 2a clinical trial testing Rho kinase (ROCK) inhibitor Bravyl (oral fasudil) in people with amyotrophic lateral sclerosis (ALS) has completed patient enrollment for its high-dose group, the company said. While the REAL trial (NTC05218668) was initially slated to only test a 180 mg daily dose of…
A new biopharmaceutical company has launched in the U.S. with more than $100 million in financing and a goal to develop a genomic medicine targeting the UNC13A protein as a novel treatment for people with amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases. Trace Neuroscience kicked off its…
Sometimes I find perspective when I listen to other rare disease communities. ALS News Today is just one publication of its parent company, Bionews, which hosts more than 50 online websites devoted to rare and chronic diseases. Last week, I got on Cystic Fibrosis News Today to catch up…
Researchers at Northwestern University received a $7.3 million research grant from the National Institute on Aging to expand their work on NU-9, which has shown promise in amyotrophic lateral sclerosis (ALS) preclinical studies, and explore its effectiveness in other neurodegenerative diseases. The institute is a division of the National…
Kadimastem, the developer of the experimental amyotrophic lateral sclerosis (ALS) therapy AstroRx, is merging with NLS Pharmaceutics to combine efforts to advance both companies’ drug portfolios. The combined company will focus on moving AstroRx and a diabetes drug being developed by Kadimastem through clinical trials. A Phase…
Mutations in the mitochondrial genome — the set of DNA instructions found inside mitochondria, the powerhouses of cells — may be linked to amyotrophic lateral sclerosis (ALS), a study found. The findings open the door for new ways of diagnosing and treating the disease, researchers said. “We aren’t saying…
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