The U.S. Food and Drug Administration (FDA) is extending by three months its review of tofersen, Biogen’s investigational treatment for forms of amyotrophic lateral sclerosis (ALS) caused by mutations in the SOD1 gene. Earlier this summer, the FDA granted the application priority review, with a decision expected no…
The nonprofit EverythingALS is teaming up with several biotech companies for a study into the feasibility of monitoring amyotrophic lateral sclerosis (ALS) progression in people via disease-specific biomarkers captured digitally at the home. The study will be open to adults with an ALS diagnosis or for whom ALS…
An extended-release formulation of PrimeC, an investigational combination therapy for amyotrophic lateral sclerosis (ALS), demonstrated a favorable pharmacokinetic profile in healthy adults in a Phase 1 trial, helping with an ongoing Phase 2b study and work into a potential pivotal trial in ALS patients. A pharmacokinetic (PK) study aims…
I’m writing this column on a tough and emotional day — the anniversary of the 2018 ALS diagnosis for my late husband, Jeff. For the day I might have planned, I’d be hiking up Old Rag Mountain in Virginia, perhaps sharing the pretty view on social media,…
Helixmith has unveiled its new multi-approach program called DART — Defeating ALS through Regenerative Therapeutics — to develop new therapies for amyotrophic lateral sclerosis (ALS). Company CEO Sunyoung Kim presented the program in person and virtually at the 2022 Cell & Gene Meeting on the Mesa, Oct. 11–13.
My 16-year-old-daughter, Sara, watched a TED Talk by Angela Duckworth on grit in her AP Language class. Sara was not impressed. “I don’t want to have grit,” she declared. Many of her high school friends are taking college math classes while she is on the standard math track. She sees…
NurOwn, Brainstorm Cell Therapeutics’ investigational cell-based therapy, leads to meaningful disease-related biomarker changes regardless of amyotrophic lateral sclerosis (ALS) severity at the start of treatment, according to data from a Phase 3 trial. Specifically, the therapy increased neuroprotection biomarker levels and anti-inflammatory molecules, and reduced those of neurodegeneration…
The COURAGE-ALS Phase 3 trial of Cytokinetic’s experimental therapy reldesemtiv in people with amyotrophic lateral sclerosis (ALS) will continue as planned following its first planned interim analysis. A data monitoring committee met to review the trial’s data so far with the goal of assessing futility, or whether the…
I love scrolling through the short videos people post on social media with tips and tricks to help make everyday tasks easier. Commonly called “hacks,” these are easy shortcuts that often involve repurposing items found around the home. For people like me who live with ALS and the…
The first patients have been dosed in a Phase 1/2a clinical study that’s testing AMDXP-2011P for its ability to trace disease-associated toxic protein clumps in the eyes of adults with amyotrophic lateral sclerosis (ALS) and Parkinson’s disease, which may allow earlier diagnosis. The study, dubbed PROBE (NCT05542576),…
