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)Â 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…
Amylyx Pharmaceuticals plans to soon apply to Health Canada, requesting approval of oral AMX0035 to treat amyotrophic lateral sclerosis (ALS) based on the findings of the CENTAUR clinical trial. The company will also explore options for early access to the therapy for patients in the country. One possibly route…
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.
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…
Under an agreement with the University of Kansas, PharmaTher has gained exclusive worldwide rights to develop and possibly commercialize ketamine as a therapy for amyotrophic lateral sclerosis (ALS). “We are pleased to have added the [ALS] program to our already impressive development pipeline…
Injecting a specific type of stem cells into the bloodstream was able to reduce muscle shrinkage and promote motor neuron survival in a mouse model of amyotrophic lateral sclerosis (ALS), a study found. According to investigators, the findings indicate these stem cells, known as multi-lineage differentiating stress enduring (Muse)…
