SOD1

Arrowhead Pharmaceuticals has filed an application in Australia seeking the green light to launch the first clinical trial of ARO-SOD1, its RNA-based therapy for amyotrophic lateral sclerosis (ALS). The therapy is designed to reduce SOD1 protein levels in ALS patients carrying SOD1 mutations, which are estimated to account…

The European Medicines Agency (EMA) has agreed to review an application seeking the approval of tofersen in treating amyotrophic lateral sclerosis (ALS) caused by mutations in the SOD1 gene, the therapy’s developer, Biogen, reported. “Today’s announcement is an important milestone for the ALS community in Europe where there is a tremendous need…

Early treatment with Biogen’s experimental therapy tofersen slows disease progression in people with amyotrophic lateral sclerosis (ALS) associated with SOD1 gene mutations, compared with patients who started treatment after a six-month delay, according to findings from a Phase 3 trial and its open-label extension study. These benefits…

Altered forms of the SOD1 protein are found in the spinal cord nerve cells of people with all types of amyotrophic lateral sclerosis (ALS), not just those with mutations in the gene providing instructions for making the protein, a study reported. “The results suggest this abnormal protein contributes to cell…

The U.S. Food and Drug Administration (FDA) has agreed to review an application from Biogen for approval of the company’s experimental therapy tofersen to treat amyotrophic lateral sclerosis (ALS) associated with mutations in the SOD1 gene. The FDA has granted the application accelerated review and a decision…

AKAVA Therapeutics’ experimental therapy AKV9, formerly NU-9, was superior to approved therapies for amyotrophic lateral sclerosis (ALS) at improving the health of lab-grown upper motor neurons from a mouse model of the disease, a study shows. Upper motor neurons are one of the two types of specialized nerve cells that…

Early use of Biogen’s tofersen significantly slows disability progression, as well as a decline in lung function, muscle strength, and quality of life in amyotrophic lateral sclerosis (ALS) patients with mutations in the SOD1 gene, compared with a six-month delay in starting treatment. These benefits were accompanied by pronounced and sustained…

A CRISPR-based gene editing system could be used to reduce the activity of genes associated with amyotrophic lateral sclerosis (ALS) and Huntington’s disease, a new study shows. The study, “Targeted gene silencing in the nervous system with CRISPR-Cas13,” was published in Science Advances. CRISPR is a strategy that…

While it’s long been believed that the death of motor neurons in the spinal cord is the main cause of amyotrophic lateral sclerosis (ALS), new research suggests that degeneration of motor neurons in the brain takes place independently and may also contribute to the disease. The study’s work in…

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.