SOD1 mutations

Arrowhead Pharmaceuticals will seek permission later this year for the first clinical trial of ARO-SOD1, its RNA-based therapy for amyotrophic lateral sclerosis (ALS), according to a company press release. Further details about Arrowhead’s plans for testing its investigational RNA therapy are expected at a company Research and…

RAG-17, an investigational therapy from Ractigen Therapeutics to treat amyotrophic lateral sclerosis (ALS), has been designated an orphan drug by the U.S. Food and Drug Administration (FDA). Orphan drug status is given to medicines intended to treat life-threatening or chronically debilitating diseases that affect fewer than 200,000 individuals…

A Phase 2 clinical trial evaluating AL-S Pharma AG’s experimental therapy AP-101 in people with amyotrophic lateral sclerosis (ALS) has enrolled its first patient. The study (NCT05039099) already is recruiting up to 63 adults with sporadic and SOD1-related familial ALS at one of its Canadian sites.

The first part of Biogen’s early access program for tofersen is now open to patients with the most rapidly progressing cases of familial amyotrophic lateral sclerosis (ALS), the company announced. These patients, with familial ALS caused by mutations in the SOD1 gene, will be the first to…

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…

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…

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…

The U.S. Food and Drug Administration (FDA) has given fast track designation to Orphazyme’s investigational therapy arimoclomol for the treatment of amyotrophic lateral sclerosis (ALS). A therapy candidate is put on the FDA’s fast…

An experimental gene therapy based on RNA interference (RNAi) shows potential to treat patients with familial amyotrophic lateral sclerosis (ALS) caused by mutations in the superoxide dismutase 1 (SOD1) gene, according to results of a preclinical study in nonhuman primates. The study, “Safe and effective superoxide…

Sensory nerves carrying mutations in the TDP-43 or SOD1 genes — associated with the development of a…