Lindsey Shapiro, PhD,  science writer—

Locanabio‘s investigational therapy significantly reduced the buildup of toxic RNA molecules in amyotrophic lateral sclerosis (ALS) patient cells and mouse models associated with C9ORF72 mutations, according to new data. These RNA products — intermediate molecules that are produced from DNA as a template for protein production — are…

WVE-004, Wave Life Sciences‘ investigational treatment for amyotrophic lateral sclerosis (ALS), significantly reduces toxic proteins associated with C9orf72 genetic mutations, but that doesn’t seem to translate into functional status gains in patients. Based on findings from the Phase 1b/2a FOCUS-C9 trial (NCT04931862), the company has decided to discontinue developing…

ATH-1105, a treatment candidate by Athira Pharma, significantly prolonged survival in a mouse model of amyotrophic lateral sclerosis (ALS), according to a company update. Consistent with previous analyses, the treatment also reduced neurodegeneration, inflammation, and the toxic buildup of the TDP-43 protein in nerve cells, which resulted…

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)…

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…

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…

A Phase 3b study evaluating the long-term safety and effectiveness of two dosing regimens of Radicava ORS (edaravone) — an approved oral formulation of the medicine — in people with amyotrophic lateral sclerosis (ALS) is now fully enrolled. The study was designed to fulfill a post-marketing commitment that…

The U.S. Food and Drug Administration (FDA) will hold an advisory committee meeting to discuss BrainStorm Cell Therapeutics‘ application to approve NurOwn for amyotrophic lateral sclerosis (ALS). Such a meeting, called an ADCOM, is normally for when a therapy is under regulatory review so experts can discuss its…

Treatment with CNM-Au8 significantly delayed clinical worsening in people with amyotrophic lateral sclerosis (ALS), according to new exploratory analyses from the therapy’s arm of the HEALEY ALS platform trial. The experimental therapy, from Clene Nanomedicine, had previously been found to reduce the risk of death by more…

Amyotrophic lateral sclerosis (ALS) patients treated earlier with CNM-Au8 in a clinical trial experienced a significantly slower disease progression than those who started treatment nine months later. These latest findings from the RESCUE-ALS Phase 2 trial (NCT04098406) and its open-label extension (OLE) study (NCT05299658) add to earlier reports that…