News

The National Organization for Rare Disorders (NORD) will celebrate the 35th anniversary of both the 1983 Orphan Drug Act and NORD’s founding at a dinner tonight in Washington, D.C. The 2018 Rare Impact Awards, to be held at the Andrew W. Mellon Auditorium, will be webcast via Facebook for those…

Transplanting engineered neural cells into the brain of an amyotrophic lateral sclerosis (ALS) animal model delayed disease progression and extended the animals’ survival, a study shows. The study, “Transplantation of Neural Progenitor Cells Expressing Glial Cell Line‐Derived Neurotrophic Factor into the Motor Cortex as a Strategy to Treat Amyotrophic Lateral…

The Muscular Dsytrophy Association (MDA) awarded 13 new grants totaling $2.6 million to find treatments for neuromuscular disorders, including amyotrophic lateral sclerosis (ALS). The 13 grants are divided into three types: Five grants were awarded to recognized, independent researchers; Five development grants were awarded to early-career scholars, and;…

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…

Mutations in the C9orf72 gene deregulate the process by which cells remove waste, leading to a buildup of toxic elements inside them, researchers have found. This discovery expands knowledge on how C9orf72 may contribute to the development and progression of neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS)…

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…