Clinigen Group recently partnered with Mitsubishi Tanabe Pharma Corporation to launch an early access program in Europe for Radicava (edaravone), an intravenous treatment for amyotrophic lateral sclerosis (ALS). The therapy is not yet approved in Europe, but the early access program will help make it available…
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…
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 European Union isn’t doing enough to protect the 30 million or so people with rare diseases who live in its 28 member countries, officials meeting last week in Vienna said. More than 900 people from 58 nations attended the 9th European Conference on Rare Diseases & Orphan Drugs (ECRD),…
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…
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