A global Phase 3 trial of AMX0035 as an oral treatment for amyotrophic lateral sclerosis (ALS) intends to enroll up to 600 patients, the therapy’s developer, Amylyx Pharmaceuticals, announced in a press release. Likely to begin in the coming months, the trial — to be called PHOENIX…
“I didn’t even know ALS existed until I was told I had it.” That’s what most patients say when telling their ALS story. I said the same thing when asked to share my story at an event in 2010, and I hear the same thing from others today, 11 years…
A Phase 2 clinical trial evaluating oral RT001 — Retrotope’s experimental synthetic fatty acid — as a treatment of amyotrophic lateral sclerosis (ALS) is fully enrolled, the company announced. The enrollment target of 40 patients was met, and exceeded, ahead of its scheduled six weeks. Dosing began…
In the summer of 2014, people across the world dumped water over their heads in the ALS ice bucket challenge and shared videos of the experience on social media. Our son, Isaac, who was 4 at the time, did it, saying, “I’m having my aunties and uncles do the…
A committee with the European Medicines Agency favors designating Seelos Therapeutics‘ investigational therapy SLS-005 (trehalose) an orphan drug as a potential treatment of amyotrophic lateral sclerosis (ALS), the company announced in a press release. The opinion, issued by the Committee for Orphan Medicinal Products (COMP), will now go to the…
Like many newly diagnosed ALS patients, I was overwhelmed by the steep learning curve of finding out more about the disease. Never mind having to jump in and help raise funds for it. Every time May rolled around with ALS Awareness Month, I was uncertain about how I fit…
National Amyotrophic Lateral Sclerosis Awareness Month, recognized in the U.S. each May since 1992, continues to unite patients, family, friends, and advocacy groups around shared goals of raising public awareness and research support. This year’s efforts continue to recognize and support those living with…
Perspective from Robert Bucelli, MD, PhD Dr. Robert Bucelli is a professor and neurologist at Washington University in St. Louis. He consults and cares for many people with ALS at the school’s world-renowned ALS Center. Dr. Bucelli is a paid consultant for Biogen and was compensated for this article.
People with very rapidly progressing familial amyotrophic lateral sclerosis (ALS) caused by mutations in the SOD1 gene may gain early access to Biogen’s experimental therapy tofersen by mid-July, the company announced. This marks the first part of Biogen’s planned access program for tofersen, and will begin after patients initially assigned…
Life is unpredictable. People get in car accidents. They have heart attacks. Tornadoes strike. I recently saw a viral video in which a rabid bobcat attacked a woman outside her home. We never really know what will happen next, and yet, we humans usually live with optimism for the…
