A Phase 3 clinical trial evaluating Orphazyme’s investigational oral therapy arimoclomol for amyotrophic lateral sclerosis (ALS) has failed to meet its primary and key secondary goals. While no safety issues were reported in the trial (NCT03491462), arimoclomol did not significantly extend patients’ lives nor…
Jan Veldink, a neurologist from the Netherlands whose work in the genetics of amyotrophic lateral sclerosis (ALS) has led to important discoveries about disease risk, won the 2021 Sheila Essey Award, according to a blog post from the ALS Association. The $50,000 award was presented at the recent…
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 combination of guanabenz — an approved therapy for high blood pressure — plus riluzole slowed disease progression in adults with early amyotrophic lateral sclerosis (ALS), particularly those with bulbar onset disease, a form of ALS, according to data from a Phase 2 clinical trial. Despite these promising benefits, the…
“Everybody’s talkin’ at me, Can’t hear a word they’re sayin’, Only the echoes of my mind.” – Fred Neil ALS has rendered me a minimalist creature of habit. My weekday routine includes the intersection of paranoid caution, practiced routine,…
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…
Registration is now open for fundraising events at four championship golf courses, whose fees will be used to fund research, care, and advocacy for neuromuscular disorders such as amyotrophic lateral sclerosis (ALS) and muscular dystrophy. According to the…
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