Voyager Therapeutics’ experimental therapy VY-SOD102 shows promise for treating patients with familial amyotrophic lateral sclerosis (ALS) caused by mutations in the superoxide dismutase 1 (SOD1) gene, according to results in animal models. The preclinical data were presented at the Congress of the European Society of Gene and Cell Therapy…
Tiglutik, an oral suspension of riluzole, is now available as a treatment for amyotrophic lateral sclerosis (ALS) in the U.S., ITF Pharma, the therapy’s developer and a subsidiary of Italfarmaco, recently announced. Rilutek (riluzole, marketed by Sanofi), has been available in the U.S. as 50 mg tablets…
SOD1 gene mutations can contribute differently to the degenerative process of nerve cells involved in the progression of amyotrophic lateral sclerosis (ALS), newly developed worm models of the disease reveal. This finding was reported in the study, “Single copy/knock-in models…
Chip technology — much like an organ-on-a-chip that can simulate the workings of tissue or organs  — was used to recreate amyotrophic lateral sclerosis (ALS), allowing researchers to identify two treatments for blood cancers — rapamycin plus bosutinib — as a possible ALS combination therapy. The study, “Microphysiological 3D model…
Researchers have discovered that mast cells and neutrophils — two types of immune cells — are involved in the degeneration of peripheral motor nerve cells and progression of amyotrophic lateral sclerosis (ALS). These findings also clarify why masitinib — an investigational therapy for ALS that targets mast cells…
The Muscular Dystrophy Association (MDA) will host its 18th annual Wings Over Wall Street benefit Oct. 17 to raise funds for amyotrophic lateral sclerosis (ALS) research with the ultimate goal of defeating the disease, the nonprofit announced. Presented by Investors Exchange and emceed by ABC affiliate…
The communication between motor neurons and muscles, which allow us to control our movements, is more dynamic than previously thought, a new study with zebra fish shows. Researchers at the Karolinska Institutet in Sweden discovered that adult motor neurons can switch their messenger, or neurotransmitter, in response to increased…
Half a year has gone by since disgraced pharma executive Martin Shkreli was sentenced to seven years in federal prison for securities and wire fraud while heading San Diego-based Retrophin. As founder and CEO of another company (then known as Turing Pharmaceuticals), in late 2015 Shkreli bought the rights…
Canadian authorities have approved the sale of Radicava (edaravone) to treat patients with amyotrophic lateral sclerosis (ALS). The therapy will be marketed by Mitsubishi Tanabe Pharma Canada (MTP-CA), a unit of Mitsubishi Tanabe Pharma America. MTP-CA was established earlier this year to distribute Radicava and other medicines for difficult-to-treat…
Personalized assessment and intervention with a specific communication strategy are key to improving communication in patients with amyotrophic lateral sclerosis (ALS). Dysarthria, the term for a group of speech disorders, affects 95% of patients with ALS. Augmentative and alternative communication (AAC), which includes aided and unaided approaches such as communication books…
