Utreloxastat (PTC857), an investigational therapy for people with amyotrophic lateral sclerosis (ALS), was safe, well tolerated, and displayed promising pharmacological properties in healthy people, according to a first-in-human Phase 1 study. Based on these findings, the therapy’s developer, PTC Therapeutics, has launched a Phase 2 trial (NCT05349721)…

Elevated levels of high-density lipoprotein (HDL) — commonly called the “good” cholesterol — are significantly associated with a poorer survival rate among people with amyotrophic lateral sclerosis (ALS), according to a population-based study in the Netherlands. In contrast, levels of total cholesterol and low-density lipoprotein…

The U.S. Food and Drug Administration (FDA) has announced an advisory committee meeting seeking advice on whether to approve Biogen‘s investigational therapy tofersen for the treatment of amyotrophic lateral sclerosis (ALS) caused by SOD1 gene mutations. The public meeting, to be held online March 22, by…

Neopharm has acquired exclusive rights to commercialize AMX0035 (sodium phenylbutyrate and taurursodiol) in Israel, Gaza, the West Bank, and to the Palestinian Authority as a treatment of amyotrophic lateral sclerosis (ALS). The company’s agreement with Amylyx Pharmaceuticals makes it responsible for the distribution and marketing of AMX0035…

An oral suspension form of Radicava (edaravone), an approved treatment of amyotrophic lateral sclerosis (ALS), was favored for patient reimbursement in Canada by a branch of the Canadian Agency for Drugs and Technologies in Health (CADTH). The opinion issued by the CADTH Canadian Drug Expert Committee recommended that Radicava Oral…

The Muscular Dystrophy Association (MDA) has invested $600,000 in PathMaker Neurosystems to help bring a potential noninvasive neuromodulator for amyotrophic lateral sclerosis (ALS) into an initial clinical trial. Funding through the MDA Venture Philanthropy (MVP) program will support an early feasibility study in a small…

Inflamed Pharma, part of the incubator and accelerator company Xlife Sciences, is partnering with researchers at the University Medical Center Gottingen in Germany to develop its investigational therapy ProcCluster for the treatment of amyotrophic lateral sclerosis (ALS). The collaboration, led by Jan C. Koch, MD, a principal investigator…

Scientists have developed a new method that allows them to grow mature nerve cells from stem cells — a technique that could be applied to study diseases like amyotrophic lateral sclerosis (ALS). “For the first time, we have been able to see adult-onset neurological protein aggregation in the stem…

Synchron‘s Stentrode, an implantable brain-computer interface designed to allow people with amyotrophic lateral sclerosis (ALS) and other paralyzing conditions to control a computer using their thoughts, showed a positive safety profile in a small clinical trial. The four people in the Australian study had ALS and severe…

Biological samples from amyotrophic lateral sclerosis (ALS) patients who took part in the Phase 3 clinical trial of NurOwn, a cell-based therapy, are being donated by BrainStorm Cell Therapeutics to a repository for use by the research community. The samples include blood and spinal fluid (cerebrospinal fluid or CSF)…