familial ALS

Researchers at the University of Texas at Dallas (UT Dallas) have received an award from the U.S. Army Medical Research Acquisition Activity to advance their preclinical research of an innovative CRISPR-based gene editing approach to treat one of the most common causes of familial amyotrophic lateral sclerosis (ALS). The two-year…

People with very rapidly progressing familial amyotrophic lateral sclerosis (ALS) caused by mutations in the SOD1 gene may gain early access to Biogen’s experimental therapy tofersen by mid-July, the company announced. This marks the first part of Biogen’s planned access program for tofersen, and will begin after patients initially assigned…

Editor’s note: The ALS News Today team is providing in-depth coverage of the 2021 Virtual AAN Annual Meeting, April 17–22. Go here to read the latest stories from the conference. More than one-quarter of amyotrophic lateral sclerosis (ALS) patients — with or without a family history of ALS — have…

Editor’s note: The ALS News Today team is providing in-depth coverage of the 2021 Virtual AAN Annual Meeting, April 17–22. Go here to read the latest stories from the conference. In a Phase 1 trial for amyotrophic lateral sclerosis (ALS), the investigational therapy AP-101 — designed to ease…

A higher than expected proportion of amyotrophic lateral sclerosis (ALS) patients — most without a family history of ALS — had mutations in genes associated with the disease, according to a study in 100 patients in the U.K. “Our study found that 42 per cent of patients involved in…

Biogen will not offer early access to tofersen, its experimental therapy for familial amyotrophic lateral sclerosis (ALS) caused by mutations in the SOD1 gene, at least until data from its pivotal clinical trial are analyzed. Now in its Phase 3 portion, the study (NCT02623699) is testing the…

A single dose of an artificial microRNA — a tiny RNA molecule that is able to control the activity of certain genes — can lower the activity of SOD1, a gene commonly mutated in patients with familial amyotrophic lateral sclerosis (ALS), a proof-of-concept study shows. According to researchers,…

Tofersen, Biogen’s investigational therapy for people with familial amyotrophic lateral sclerosis (ALS) caused by SOD1 gene mutations, was safe and generally well-tolerated over three months, and appeared to lower SOD1 protein levels in the central nervous system, a Phase 1/2 clinical trial has found. There…

Biotech company EnClear Therapies has announced $10 million in financing to help advance its proprietary platform for stopping the progression of neurodegenerative disorders, including amyotrophic lateral sclerosis (ALS). The investments comes from a Series A financing round, which is part of an early development stage for start-up…

A single spinal injection of an investigational RNA therapy blocked motor neuron degeneration and saved motor function in animal models of familial amyotrophic lateral sclerosis (ALS) linked to SOD1 mutations, a study  reports. Giving the treatment — a virus-delivered gene-silencer targeting the SOD1 gene —  before the onset of…