Voyager Therapeutics presented new data from a study in an animal model of its clinical candidate VY-SOD101, showing the therapy is effective as it targets the most common cause of familial amyotrophic lateral sclerosis (ALS). The presentation, along with another preclinical program data presentation on a Huntington’s disease therapy…
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A new mouse model developed by researchers at Karolinska Institute in Sweden revealed a molecular mechanism that may contribute to the development of neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS) or multiple sclerosis. The mouse model was described in the study, “Fatal demyelinating disease…
The protein TRIF can prevent resident immune cells of the brain from activating abnormally and slow the progression of amyotrophic lateral sclerosis (ALS), researchers in Japan working in a mouse model of the disease report. Their work, “Innate immune adaptor TRIF deficiency accelerates disease progression of ALS mice with…
The distinct processes that drive FUS proteins to aggregate and clump in both amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) were detailed in a new report. The study, “Phase Separation of FUS Is Suppressed by Its Nuclear Import Receptor and Arginine Methylation,” appeared in the journal Cell. The…
Worldwide Clinical Trials and the healthcare technology company Datavant are partnering in an effort to improve trials in neurological diseases such as amyotrophic lateral sclerosis (ALS). With the aging of Western populations, neurodegenerative diseases such as ALS, Alzheimer’s, Parkinson’s and Huntington’s represent an increasingly high…
Biohaven Pharmaceuticals has established an expanded access program (EAP) for patients with amyotrophic lateral sclerosis (ALS) for its investigational treatment candidate, BHV-0223, the company recently announced. The United States Food and Drug Administration regulates the expanded access program to help provide access to therapies that are currently in development and have…
Newly developed molecular treatment candidates called small interfering RNAs (siRNAs) have been successfully delivered to the brain and spinal cord in a rat preclinical study, Alnylam Pharmaceuticals recently announced. According to the company, the development of siRNAs may lead to the prevention or treatment of diseases such as amyotrophic…
Transplanting human spinal cord-derived neural stem cells (HSSC) into the spine of patients with amyotrophic lateral sclerosis (ALS) was found to improve their survival and function, according to clinical trial investigators. Results from the Phase 1/2 trials were compared to historical populations (controls), and the results were reported in the…
The Muscular Dystrophy Association (MDA) recently awarded the Massachusetts General Hospital (MGH) a $750,000 human clinical trial grant to study positron emission tomography (PET) imaging to measure inflammation in amyotrophic lateral sclerosis (ALS) patients as a potential biomarker. This grant, which is part of a nonprofit partnership called…
Patients with amyotrophic lateral sclerosis (ALS) show high prevalence of hypermetabolism, which leads to more rapid physical decline and earlier death, new study reports. The research, “Hypermetabolism in ALS is associated with greater functional decline and shorter survival,” appeared in the Journal of Neurology, Neurosurgery & Psychiatry.