News

Inhibition of a protein called Cdk5 in the brain increases lifespan and improves motor function in mice with amyotrophic lateral sclerosis (ALS), a new study reports. This evidence supports a potential role for Cdk5 in the progression of ALS and opens new avenues as a potential therapeutic target for…

Rare diseases deeply affect not only the children who experience them, but also their healthy brothers and sisters, as their parents can attest.    Two entries in November’s “Disorder: The Rare Disease Film Festival” will focus on what siblings go through, according to the San Francisco festival’s co-founder,…

Disarm Therapeutics has announced the publication of two studies that further researchers’ understanding of the structure and function of SARM1, a key protein in cellular degeneration. The new data may aid in the design of therapies for multiple diseases, including amyotrophic lateral sclerosis (ALS). The findings were published in…

Orphazyme announced that it and Worldwide Clinical Trials will continue their collaboration through the long-term extension of a Phase 3 study into arimoclomol, a potential oral treatment for amyotrophic lateral sclerosis (ALS). ORARIALS-01 is a randomized, placebo-controlled and double-blind trial (NCT03491462) underway at 30 centers across North America…