The recent conditional approval of Qalsody (tofersen) for adults with amyotrophic lateral sclerosis (ALS) who carry mutations in the SOD1 gene is a major cause for celebration, according to the Muscular Dystrophy Association (MDA). The therapy earned accelerated approval from the U.S. Food and Drug Administration (FDA)…

A Phase 2b trial that’s testing PrimeC in people with amyotrophic lateral sclerosis (ALS) has reached 80% enrollment and is on track to complete patient recruitment by June, NeuroSense Therapeutics announced in a quarterly update. Called PARADIGM (NCT05357950), the study began enrolling ALS patients in…

Radicava (edaravone) significantly prolongs long-term survival for people with amyotrophic lateral sclerosis (ALS), according to an analysis of nearly 3,000 patients. It didn’t significantly slow the progression of disease symptoms, as assessed by scores on the ALS Functional Rating Scale-Revised (ALSFRS-R), however. The results were presented at last…

Daily oral treatment with memantine did not slow disease progression nor did it reduce biomarker changes among people with amyotrophic lateral sclerosis (ALS) enrolled in a Phase 2b trial. Approved under the brand name Namenda to treat cognitive problems in people with Alzheimer’s disease, memantine has also…

It’s been nearly three years since the HEALEY platform clinical trial started and, so far, two promising experimental treatments for amyotrophic lateral sclerosis (ALS) have been identified. Both potential medications — CNM-Au8 and pridopidine — are headed toward Phase 3 clinical testing, while testing has been ruled…

Treatment with the investigational therapy DNL343 was generally well-tolerated among people with amyotrophic lateral sclerosis (ALS) in an early clinical trial, and biomarker data from the trial suggest that the therapy is working as intended. Results were presented at the annual meeting of the American Academy of Neurology (AAN),…

The U.S. Food and Drug Administration (FDA) has granted conditional approval to Biogen’s tofersen, now named Qalsody, for the treatment of amyotrophic lateral sclerosis (ALS) associated with mutations in the SOD1 gene. The decision, which marks the first conditional approval for ALS in the country, comes about eight…

A new technology powered by artificial intelligence (AI) may one day help doctors diagnose people in the early stages of amyotrophic lateral sclerosis (ALS), according to data from a clinical trial. Developed by Pison, the technology was able to detect involuntary muscle twitches, or fasciculations, and changes in…

Arrowhead Pharmaceuticals will seek permission later this year for the first clinical trial of ARO-SOD1, its RNA-based therapy for amyotrophic lateral sclerosis (ALS), according to a company press release. Further details about Arrowhead’s plans for testing its investigational RNA therapy are expected at a company Research and…

An immune protein that is mostly produced in neurons was found to drive damage to mitochondria, which produce energy for cells, and nerve fiber loss, a study has found. Suppressing the protein, called gasdermin-E (GSDME), prevented nerve fiber loss in cells derived from people with amyotrophic lateral sclerosis (ALS),…