Margarida Maia, PhD,  science writer—

Quralis has completed dosing in the first group of healthy adults who volunteered for its Phase 1 clinical trial of QRL-101, an experimental oral small molecule being developed for amyotrophic lateral sclerosis (ALS). QRL-101-03 (NCT06532396) is expected to enroll about 60 volunteers who’ll be divided into five…

The U.S. Food and Drug Administration (FDA) has given the green light to Dr.Noah Biotech to initiate a Phase 1 clinical trial of NDC-011, a combination therapy for amyotrophic lateral sclerosis (ALS), in healthy volunteers. The trial, planned for the first half of 2025 at a single site…

A strategy that simultaneously targets motor neurons, astrocytes, and microglia — three cell types involved in the development and progression of amyotrophic lateral sclerosis (ALS) — was shown to further extend survival in a mouse model of the disease. Specifically, targeting the three cell types at the same time…

Monepantel, a veterinary drug that PharmAust is repurposing for amyotrophic lateral sclerosis (ALS), continues to slow disease progression and extend survival when taken daily for up to nearly two years, while being generally well tolerated by patients. That’s according to interim results from an open-label extension…

The Muscular Dystrophy Association (MDA) is accepting applications for its Advocacy Collaboration Grant program, which supports projects from organizations working to improve the lives of people with neuromuscular diseases such as amyotrophic lateral sclerosis (ALS).  Applications for this year’s program are open until Oct. 18, and…

A decade later, the family of Pete Frates, the amyotrophic lateral sclerosis (ALS) patient who helped start the Ice Bucket Challenge, is relaunching the campaign that went viral on social media, raising awareness and money to support ALS research. In an event organized by the Peter Frates…

Nearly a year of treatment with TPN-101, an investigational oral molecule from Transposon Therapeutics, safely slowed disease progression and lung function decline in people with amyotrophic lateral sclerosis (ALS) related to C9orf72 mutations. That’s according to final data from a Phase 2a study (NCT04993755) that tested TPN-101…

People with amyotrophic lateral sclerosis (ALS) who received one year of treatment with PrimeC, an experimental oral tablet from NeuroSense Therapeutics, lived significantly longer without disease complications and had a slower lung function decline than those who started treatment six months later. That’s according to the latest results…

Elevated blood levels of neurofilament light chain (NfL), a biomarker of nerve cell damage, are linked to faster disease progression and shorter survival in most people with amyotrophic lateral sclerosis (ALS), a new study found. However, the team of researchers observed that blood NfL levels among patients differed by…

An advisory committee of the European Medicines Agency (EMA) has recommended against granting conditional marketing authorization to masitinib as an oral add-on treatment for amyotrophic lateral sclerosis (ALS). The negative opinion by the Committee for Medicinal Products for Human Use (CHMP) confirms the committee’s view, announced last…