The first patient has been enrolled in a Phase 2a clinical trial investigating Helixmith’s gene therapy Engensis (VM202) for the treatment of amyotrophic lateral sclerosis (ALS), the company announced. The REViVALS-1A study (NCT04632225) intends to recruit 18 patients across four clinical sites in the U.S. Enrollment is open…
Editor’s note: The ALS News Today team is providing in-depth coverage of the 2021 MDA Virtual Clinical and Scientific Conference, March 15–18. Go here to read the latest stories from the conference. Variants in the WWOX gene may contribute to the development of amyotrophic lateral sclerosis (ALS)…
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 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…
“‘Cause I got a peaceful, easy feelin’, And I know you won’t let me down, ‘Cause I’m already standin’ On the ground.” Those lyrics by Jack Tempchin, sung by the Eagles on their…
“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…
Changes to a critical structure within the cell nucleus of spinal nerve cells — the nucleolus — preceded the appearance of other disease markers in people with either familial or sporadic forms of amyotrophic lateral sclerosis (ALS), a study has discovered. …
Many diseases have their own awareness color — breast cancer is pink, muscular dystrophy is green, and AIDS is red, for example — but what’s the significance of pink, green, blue, and purple lights side-by-side? These are the colors most often used to represent Rare Disease Day. This…
“I’m glad I got the chance to miss you,” I told my husband, Todd, when I got home after a day trip skiing with our children last week. Since Todd has ALS and I am his caregiver, we rarely spend time apart. Now that he is paralyzed, we don’t…
The small difference in treatment responses among amyotrophic lateral sclerosis (ALS) patients receiving NurOwn versus a placebo in a recent clinical trial likely was due to chance, the U.S. Food and Drug Administration (FDA) announced in a press release. Along with slightly more deaths in the NurOwn…
