#MDA2021 – Rare WWOX Mutations in Patients Affect Mitochondria of Cells

HEALEY Trial Reaches 50% Enrollment for First 3 Regimens

The HEALEY ALS platform trial, which is evaluating the safety and efficacy of several potential treatments for amyotrophic lateral sclerosis (ALS), has passed the 50% participant enrollment target for its first three regimens. The investigational treatments are UCB’s zilucoplan, Biohaven Pharmaceuticals’ verdiperstat, and Clene Nanomedicine’s CNM-Au8. A fourth candidate…

Mitsubishi Tanabe Establishes New US Research Facility

Mitsubishi Tanabe Pharma Corporation (MTPC) is opening a new research site in the Boston area dedicated to discovering new therapeutic targets in amyotrophic lateral sclerosis (ALS) and other diseases of the central nervous system. Located in the SmartLabs biotechnology hub in Cambridge, Massachusetts, the company’s NeuroDiscovery Lab is scheduled…

Partnership Gets Communication Devices to Mississippi ALS Patients

Under a new local partnership, Mississippi residents who have amyotrophic lateral sclerosis (ALS) will have free access to communication devices and training. The program is a collaboration between speech-language graduate students at the Mississippi University for Women’s Speech & Hearing Center and the ALS Association Louisiana-Mississippi Chapter.

With Clinical Trial Criteria, We’re Stuck on the Outside, Looking In

“Have you ever participated in clinical trials?” That’s a common question among ALS patients. What’s my standard answer? “It’s complicated. I’m participating in several studies, but even though I’d like to, I haven’t been in any clinical trials. I’m excluded because I’ve had ALS for too many years.” ‘What? Tell…