functional decline

While women with amyotrophic lateral sclerosis (ALS) see a faster functional decline, men have shorter survival times, mostly due to more rapid weight loss and lung function worsening, a study shows. The findings highlight the importance of considering sex-specific differences when studying the mechanisms leading to ALS and when…

Early declines in neurofilament light chain (NfL), a biomarker of nerve cell damage, after treatment with tofersen may predict a slower disease progression over time in people with SOD1-associated amyotrophic lateral sclerosis (ALS). Patients who received tofersen early also experienced a significantly slower worsening of clinical function than…

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…

A decision is expected by May from the U.S. Food and Drug Administration (FDA) on an application seeking approval of an oral formulation of edaravone (MT-1186) — one with a similar clinical profile to Radicava — as a treatment for amyotrophic lateral sclerosis (ALS). The FDA is now…

Amylyx Pharmaceuticals has filed an application with the European Medicines Agency seeking the approval of its lead candidate AMX0035 as a treatment of amyotrophic lateral sclerosis (ALS), the company announced in a press release. The marketing authorization request follows similar submissions to Health Canada and…

Amylyx Pharmaceuticals is seeking approval in the U.S. for AMX0035, its investigational therapy for slowing functional decline in people with amyotrophic lateral sclerosis (ALS). The company submitted a new drug application (NDA) to the U.S. Food and Drug Administration (FDA) for regulatory review. The decision had been…

Treatment with AMX0035, Amylyx’s experimental oral therapy, significantly extends the lives of amyotrophic lateral sclerosis (ALS) patients with rapidly progressing disease, according to a long-term survival analysis of the CENTAUR Phase 2/3 trial. The new data were reported in a study titled “Long‐Term…

IPL344, Immunity Pharma‘s investigational therapy for amyotrophic lateral sclerosis (ALS), has a positive safety and tolerability profile and slows disease progression by about 50%, according to interim results from a small Phase 1/2a clinical trial. The company said it now is planning a placebo-controlled trial to evaluate IPL344…

Experimental treatment reldesemtiv appears to reduce functional deterioration in patients with fast progressing amyotrophic lateral sclerosis (ALS), a new analysis of the FORTITUDE-ALS trial shows. The additional analyses were presented by the trial’s principal investigator, Jeremy M. Shefner, MD, PhD, a professor at the Barrow Neurological Institute…

Patients with amyotrophic lateral sclerosis (ALS) show high prevalence of hypermetabolism, which leads to more rapid physical decline and earlier death, new study reports. The research, “Hypermetabolism in ALS is associated with greater functional decline and shorter survival,” appeared in the Journal of Neurology, Neurosurgery & Psychiatry.