Margarida Maia, PhD,  science writer—

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…

Treatment with fasudil (RT1968), which Raya Therapeutic is testing for amyotrophic lateral sclerosis (ALS), was safe and outperformed a placebo at preserving motor neurons in adults with early-stage disease. That’s according to data from ROCK-ALS (NCT03792490), an investigator-led Phase 2a trial that tested how safe fasudil is…

An advisory committee of the European Medicines Agency (EMA) is leaning against recommending the conditional approval of AB Science’s masitinib as an oral add-on treatment for amyotrophic lateral sclerosis (ALS), the company has announced in a press release. The Committee for Medicinal Products for Human Use (CHMP)…

Clene Nanomedicine has nearly doubled the enrollment cap for a soon-to-launch expanded access program (EAP) to allow more patients to receive CNM-Au8 — its investigational treatment for amyotrophic lateral sclerosis (ALS) — outside of clinical trials. Also known as a compassionate use program, the EAP will…

The Les Turner ALS Foundation, in an effort to boost and diversify clinical trial participation, has written a step-by-step guide to inform people on why clinical research in amyotrophic lateral sclerosis (ALS) needs a diverse pool of participants, how to join clinical research, and what to expect when…

People with amyotrophic lateral sclerosis (ALS) who continued treatment with NurOwn (debamestrocel) after completing a Phase 3 trial experienced additional benefits with extended treatment, data show. The findings come from 10 adults who received up to six additional doses of NurOwn over nearly one year in an expanded…