Steve Bryson, PhD, science writer —

Treatment with Qalsody (tofersen) in the real world slowed disease progression in people with amyotrophic lateral sclerosis (ALS) caused by SOD1 mutations, a small study reports. It also stabilized patients’ quality of life and lowered levels of nerve damage-related biomarkers, which is consistent with clinical trial data that…

The developer of the add-on treatment masitinib for amyotrophic lateral sclerosis (ALS) has been given the go-ahead to ask Health Canada to reconsider its decision from earlier this year against the approval of the oral therapy. The Canadian regulatory agency granted reconsideration eligibility to AB Science for…

Relyvrio (sodium phenylbutyrate and taurursodiol), an approved treatment for amyotrophic lateral sclerosis (ALS), was voluntarily removed from the U.S. and Canadian markets. Amylyx Pharmaceuticals, the therapy’s developer, made the decision after top-line results from the Phase 3 PHOENIX trial (NCT05021536) showed that patients who received Relyvrio…

More than a third of people diagnosed with amyotrophic lateral sclerosis (ALS) also showed signs of frontotemporal dementia (FTD), according to a new study of brain tissue. In more than 90% of tissues examined, patients had clumps of the TDP-43 protein, a hallmark of…

AcuraStem has secured nearly $7 million in grant funding from the National Institutes of Health (NIH) and the Department of Defense (DOD) to accelerate the development of therapies for amyotrophic lateral sclerosis (ALS) and related diseases. Coming on the heels of a…

Cigarette smoking, either currently or in the past, significantly increases the likelihood of developing amyotrophic lateral sclerosis (ALS), particularly for women, according to a pooled meta-analysis of 32 studies. A risk of the neurodegenerative condition was higher in current smokers, and it increased in a non-linear manner with more…

The ALS Society of Canada (ALS Canada) has awarded two expedited grants totaling $200,000 to international researchers seeking to advance understanding of amyotrophic lateral sclerosis (ALS). Provided through the 2023 ALS Canada Acceleration Grant Program, a $100,000 grant will support Mónica Povedano, MD, at the Hospital de Bellvitge-IDIBELL…

A European Medicines Agency (EMA) committee again has delayed issuing an opinion on whether to grant conditional approval to masitinib, a potential add-on oral therapy for amyotrophic lateral sclerosis (ALS). AB Science, the therapy’s developer, now expects a recommendation in the second quarter of this year. The…

The proportion of people diagnosed with familial amyotrophic lateral sclerosis (ALS) among all cases is 8%, according to a pooled analysis of studies published worldwide. Still, data show there was a wide variation of estimates across the studies based partly on geographical differences, study design, the definition of familial…

A previously unknown mutation in the SPTLC2 gene was identified in two patients who developed juvenile-onset amyotrophic lateral sclerosis (ALS), a study reports. The mutation significantly increased the production of certain types of fat-like molecules called sphingolipids, resulting in early-onset muscle weakness, progressive motor impairment, and involuntary tongue movements.