Genetic mutations in two previously unrecognized genes, the microtubule-associated protein tau (MAPT) and BNIP1 genes, are associated with elevated risk for amyotrophic lateral sclerosis (ALS). The study with that finding “Selective genetic overlap between amyotrophic lateral sclerosis and diseases of the frontotemporal dementia spectrum,” was published in the…
A little-known government entity within the National Institutes of Health (NIH) is helping to lead U.S. efforts to speed up the development of therapies for some 7,000 rare diseases. The Office of Rare Diseases Research (ORDR), headquartered in Bethesda, Maryland, was established in 1993 within the NIH Office of the…
NMD Pharma has obtained $47 million in financing from new investors to continue developing treatments for ALS and other neuromuscular disorders. The Danish company is focusing on small-molecule inhibitors of a muscle-related chloride ion channel. It has created a screening platform to identify potential inhibitors of the ClC-1 ion channel.
Health Canada accepted Mitsubishi Tanabe Pharma’s new drug submission (NDS) for edaravone as an intravenous treatment option for amyotrophic lateral sclerosis (ALS). Edaravone is currently approved as a treatment for ALS in Japan, South Korea, and the United States (where it is marketed by Mitsubishi Tanabe as Radicava).
Rilutek (riluzole) extends survival of patients with amyotrophic lateral sclerosis (ALS) who are at advanced stages of the disease, rather than preventing disease progression is early stages. Those findings were reported by a team led by King’s College London researchers and published in The Lancet Neurology. The…
AveXis, which is running clinical trials of a promising gene therapy for spinal muscular atrophy (SMA) — and developing a potentially similar therapy for a genetic form of amyotrophic lateral sclerosis (ALS) — announced plans to be acquired by Novartis for $8.7 billion in cash. AVXS-101 is the…
Lack of an enzyme prevents movement nerve cells from developing properly, suggesting that the shortage may play a role in ALS, a Northwestern University study reports. The study in the journal Cell Stem Cell was titled “Dissecting the Functional Consequences of De Novo DNA Methylation Dynamics in Human Motor Neuron Differentiation and…
Biotech investors MP Healthcare Venture Management (MPH), Amgen Ventures, and Alexandria Venture Investments have joined efforts by the new private biotech company QurAlis to search for a cure for amyotrophic lateral sclerosis (ALS). Research has shown that ALS is a spectrum of disorders with diverse underlying mechanisms. Similar to…
The first patients to complete a Phase 2 clinical trial have chosen to continue treatment in an open-label extension (OLE) study of Amylyx Pharmaceuticals‘ investigative therapy AMX0035 for the treatment of amyotrophic lateral sclerosis (ALS). AMX0035 is an oral combination of two small molecules, sodium phenylbutyrate (PB) and tauroursodeoxycholic acid…
Higher levels of a specialized type of immune cell may help halt the progression of amyotrophic lateral sclerosis (ALS), a study in humans and mice found. These findings were reported in the journal JAMA Neurology, in the study, “Association of Regulatory T-Cell Expansion With Progression of Amyotrophic…
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