News

SBT-272, in Phase 1 Trial, Continues to Protect Motor Neurons in Mice

Stealth BioTherapeutics’ investigational therapy SBT-272 eased inflammation and upper motor neuron degeneration in a mouse model of amyotrophic lateral sclerosis (ALS) associated with the accumulation of toxic TDP-43 protein clumps, the company announced. Motor neurons are nerve cells that control voluntary movement and progressively die in people with ALS.

B12 Slows Functional Decline in Early-stage ALS, Data Show

Treatment with ultrahigh-dose methylcobalamin — the physiologically active form of vitamin B12 — effectively slowed functional decline in people with early-stage amyotrophic lateral sclerosis (ALS) and moderate progression, data from a small Phase 3 trial in Japan show. The findings confirm previous results from a Phase 2/3 trial and…

NU-9 Is Now AKV9, and Doing Well in Preclinical Studies

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…

CNM-Au8 Extends Survival, RESCUE-ALS Study Suggests

Treatment with the experimental gold nanocrystal therapy CNM-Au8 decreased the risk of mortality by 64% among people with early amyotrophic lateral sclerosis (ALS), compared to what would be expected without treatment, according to new analyses from the RESCUE-ALS clinical trial. The findings were presented by Clene Nanomedicine,…

Starting Tofersen Early Slows ALS Progression Better: Trial Data

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…