Patricia Inácio, PhD,  science writer—

The first patient has been enrolled in a Phase 2a clinical trial investigating Helixmith’s gene therapy Engensis (VM202) for the treatment of amyotrophic lateral sclerosis (ALS), the company announced. The REViVALS-1A study (NCT04632225) intends to recruit 18 patients across four clinical sites in the U.S. Enrollment is open…

Editor’s note: The ALS News Today team is providing in-depth coverage of the 2021 MDA Virtual Clinical and Scientific Conference, March 15–18. Go here to read the latest stories from the conference. Variants in the WWOX gene may contribute to the development of amyotrophic lateral sclerosis (ALS)…

Canadian authorities have approved Xeomin (incobotulinumtoxinA) for the treatment of chronic sialorrhea, or excessive drooling, associated with neurological diseases such as amyotrophic lateral sclerosis (ALS). The decision makes Xeomin the first and only neurotoxin for chronic sialorrhea licensed for this indication in Canada. The therapy was…

A first patient has been dosed in the Phase 2 trial investigating pegcetacoplan (APL-2), Apellis Pharmaceuticals‘ candidate therapy for amyotrophic lateral sclerosis (ALS), the company and the Swedish Orphan Biovitrum (Sobi) announced. The potentially pivotal trial, called MERIDIAN (NCT04579666), is currently enrolling patients…

The ALS Association and I AM ALS have filed a petition with the U.S. Food and Drug Administration (FDA), calling on the regulatory agency and Amylyx Pharmaceuticals to make AMX0035 available to people with amyotrophic lateral sclerosis (ALS) as quickly as possible. Signed by more than 50,000 patients, their caregivers and family, the…

Scribe Therapeutics and Biogen, a biotechnology company focused on neurological diseases, have established a research partnership to develop new gene therapies for amyotrophic lateral sclerosis (ALS) using the gene-editing technology CRISPR-Cas9. The collaboration will focus initially on the development and commercialization of CRISPR-based therapies that address some…

The first patient has been dosed in a Phase 2a clinical trial evaluating the safety and effectiveness of ALZT-OP1a, a dry-powder inhaled formulation of cromolyn, in people with mild-to-moderate amyotrophic lateral sclerosis (ALS). The trial (NCT04428775) is recruiting up to 80 patients, ages 18–75, who have been…

The ALS Association and I AM ALS have opened a petition calling for the earliest possible approval of Amylyx’s experimental oral treatment AMX0035, after a Phase 2/3 trial found the therapy to be safe and to slow functional decline in amyotrophic lateral sclerosis (ALS) patients with rapidly progressing disease. Addressing the…

The Phase 3 clinical trial testing an oral suspension formulation of edaravone for amyotrophic lateral sclerosis (ALS) has resumed dosing and patient recruitment, its developer, Mitsubishi Tanabe Pharma America (MTPA), announced. The trial (NCT04165824) was launched in November, but was paused in March due to the COVID-19 pandemic. The study,…

Researchers report having created a new human-on-a-chip model, one able to more accurately simulate the clinical features of amyotrophic lateral sclerosis (ALS) in people. The chip uses motor neurons — the nerve cells responsible for controlling voluntary muscles — derived from stem cells isolated from ALS patients. This approach…