Phase 3 Trial of Ultomiris in ALS Patients Stopping for Lack of Efficacy

Alexion Pharmaceuticals is stopping further work on Ultomiris (ravulizumab) — its approved therapy for complement-associated rare blood disorders — as a potential treatment of amyotrophic lateral sclerosis (ALS) based on interim data from the CHAMPION-ALS Phase 3 clinical trial. While top-line results were expected by mid-2022, the study’s independent data…

People affected by amyotrophic lateral sclerosis (ALS) have come up with plenty of creative ways to raise money for research and care — whether it’s the ALS Ice Bucket Challenge, re-creating Olympic events, or Major League Baseball’s designation of June 2 as Lou Gehrig Day. Joe…

MediciNova is still recruiting adults with early-stage amyotrophic lateral sclerosis (ALS) for the COMBAT-ALS Phase 2b/3 clinical trial evaluating its investigational oral therapy MN-166 (ibudilast). Patients are being enrolled at 19 sites across North America; more information about contacts and locations is available here. “We continue to…

I pleaded. I bargained. I threatened. But it was no use. My dog would not back away from the woods behind the shed, where he had been sprayed by a skunk just two weeks earlier. I was more panicked when he got skunked than I was now. I frantically searched…

Clene, and its wholly owned subsidiary Clene Nanomedicine, have signed two lease agreements that will more than quadruple the manufacturing capacity of CNM-Au8, their investigational therapy for people with amyotrophic lateral sclerosis (ALS). The first lease is for a 10-year agreement for a 74,210-square-foot building in Elkton, Maryland.

Long-term treatment with Radicava (edaravone) is generally safe and nearly doubles survival time in adults with amyotrophic lateral sclerosis (ALS), according to a small real-life study in Japan. These data add to increasing, but still limited, evidence showing the therapy prolongs the life of ALS patients and…

This past week was filled with voices. No, I wasn’t hearing voices in my head. But everything seemed to involve a voice. Let me explain. An action hero uses his voice I watched the newly released documentary “Val,” currently streaming on Amazon Prime Video. By the time the final…

Completing their version of 102 Olympic events — including archery, diving, and fencing — in 17 days, Stuart Bates and Charlotte Nichols raised some £150,000 (about $207,000) for the U.K.’s Motor Neurone Disease (MND) Association. The Oxfordshire duo wound up their grueling challenge, thought to be the first of…

Registration is now open for Global Genes‘ 2021 RARE Patient Advocacy Summit. This year’s hybrid event will be livestreamed from California Sept. 27-29, and some seats also are available for attending the event in person in San Diego. “Here you’ll have the opportunity to connect and engage with others…

Defects in cholesterol metabolism may account for some of the neurological damage that occurs in amyotrophic lateral sclerosis (ALS) and other diseases marked by problems with the protein TDP-43, a study reported. As cholesterol  is crucial to nerve cells, these results suggest that restoring its metabolism may be a…