Repeated muscle injections with Engensis (VM202), Helixmith’s investigational non-viral gene therapy, were generally safe and well-tolerated in people with amyotrophic lateral sclerosis (ALS), according to top-line data from a Phase 2a clinical trial. While the sample size was too small to determine the therapy’s efficacy, muscle biopsies were…
Whole-genome sequencing, a type of genetic screening that examines a person’s complete DNA sequence, may be a promising way to identify disease-causing mutations in people with amyotrophic lateral sclerosis (ALS), according to a study of Italian patients. Disease-associated mutations were found in more than a quarter of 1,043 Italian…
Amydis is developing a test to monitor the buildup of TDP-43 in the eye’s retina of people with amyotrophic lateral sclerosis (ALS), which could offer a noninvasive way to diagnose the neurodegenerative disease sooner and better monitor its progression. The research will be conducted with support from a…
A number of stem cell lines developed to better understand how certain genetic factors might contribute to amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases is now available to scientists worldwide. The cell lines were engineered by researchers at the National Institutes of Health Center for Alzheimer’s and Related…
Real-world use of Radicava (edaravone) significantly prolongs survival in amyotrophic lateral sclerosis (ALS) patients, with people in the U.S. on the therapy living about six months longer than those not on this treatment, an analysis of Radicava’s use in clinical settings reported. The estimated probability of survival was greater…
Long-term use of Radicava (edaravone) is safe and may modestly slow disease progression in people with amyotrophic lateral sclerosis (ALS), according to a small Korean study. Patients also experienced no changes in phrenic nerve function, which is required for the contraction and expansion of the diaphragm muscle. “Further…
A protein called NOVA1 may be involved in the early disruption of protein production in amyotrophic lateral sclerosis (ALS), even before the characteristic buildup of the toxic TDP-43 protein occurs. The finding is a step toward identifying ways to treat the disease in its earlier stages, before nerve cells…
Amylyx Pharmaceuticals has partnered with Sunnybrook Research Institute (SRI) to find new therapeutic candidates for amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases. Under terms of the two-year sponsored research agreement, SRI will work to identify potential therapeutics that potently and specifically inhibit Bax and Bak, two proteins…
A U.S. Food and Drug Administration (FDA) advisory committee will meet  Sept. 7 to discuss the approval of AMX0035 for the treatment of amyotrophic lateral sclerosis (ALS), Amylyx Pharmaceuticals announced. While the application was granted priority review earlier this year, with a final decision expected by June…
An at-home music therapy program aimed at improving speech, swallowing, and respiration in people with amyotrophic lateral sclerosis (ALS) was well-tolerated and feasible, according to a small pilot study. While the pilot study (NCT03604822) was not designed to test the program’s effectiveness, collected data suggest that the music…
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