Ubiquitination — the cell’s process of tagging proteins, often to degrade unwanted ones — is essential for the disassembly of stress granules that, by not being cleared, damage cells in amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases, a study showed. The study, “Ubiquitination is essential for…

Coya Therapeutics’ ALS001, a potential regulatory T-cell (Treg) therapy designed to halt amyotrophic lateral sclerosis (ALS) progression, has been granted an orphan drug designation by the U.S. Food and Drug Administration (FDA). Furthermore, a Phase 2a trial (NCT04055623) testing ALS001 versus a placebo in 12 ALS patients…

Using motor neurons derived from people with amyotrophic lateral sclerosis (ALS) is a promising approach to screen for molecules that prevent certain disease mechanisms, and to identify new targets for therapies, according to a recent study. Two classes of therapies were identified that might lessen the excessive firing of motor…

A July 15 golfing event brings the ALS Therapy Development Institute (ALS TDI) together with he Azzur Group to raise money to help support research into potential new treatments for amyotrophic lateral sclerosis (ALS). The Azzur Fore ALS Golf Tournament is set to take place on Thursday, July…

A branch of the European Medicines Agency (EMA) has recommended that Prilenia’s investigational therapy pridopidine be designated an orphan medicine to treat people with amyotrophic lateral sclerosis (ALS). Medicines with the potential to become safe and effective treatments for rare, life-threatening, or chronically debilitating conditions affecting no more than one…

Note: This story was updated June 30, 2021, to clarify that Exservan is placed on top of the tongue, not under it. Specialty pharmacy services for Exservan (riluzole), a twice-daily film that dissolves when placed on the tongue and approved to treat amyotrophic lateral sclerosis…

A previously unknown form of amyotrophic lateral sclerosis (ALS) — one with onset during childhood — is caused by mutations that alter the production of certain lipids (fat molecules), scientists report. “We found that a genetic form of the disease can also threaten children. Our results show for the first…

Cedars-Sinai Medical Center, in California, has been awarded an $11.99 million grant to support a clinical trial that will test specifically engineered neural progenitor cells as a potential therapy for amyotrophic lateral sclerosis (ALS). The work, funded by the California Institute for Regenerative Medicine, or CIRM, will build…

In a response letter to The ALS Association, the U.S. Food and Drug Administration (FDA) has recognized the unmet therapeutic need of people with amyotrophic lateral sclerosis (ALS) and reaffirmed its commitment to the 2019 ALS clinical trial guidance. However, the agency did not provide any details about how it…

Largely due to going virtual this year, the annual HayesDavidson 5K (HD5K) charity run met its goal of raising £40,000 (nearly $56,000) for the Motor Neurone Disease (MND) Association. The event previously was held in Hyde Park in London. However, the switch to the digital format enabled more…