AB Science’s proposed measures to ensure patient safety in clinical trials of its experimental oral therapy masitinib were deemed sufficient by the French National Agency for Medicines and Health Products Safety (ANSM), and patient enrollment in the country may resume. The announcement comes less than two months after…
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In keeping with its commitment to turn amyotrophic lateral sclerosis (ALS) into a “livable disease” by 2030, and to ramp up discovery and funding of new therapy candidates, the ALS Association is moving to a unified structure from a federated one. “Our best opportunity to fulfill this promise and…
Verge Genomics and Eli Lilly have entered into a three-year collaboration to find and develop therapies for amyotrophic lateral sclerosis (ALS). Verge will focus on discovering and validating potential new therapeutic targets, while Lilly will select up to four candidates to advance through clinical testing with a goal of…
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Prilenia’s pridopidine for the treatment of amyotrophic lateral sclerosis (ALS). The decision follows a recent positive opinion from a branch of the European Medicines Agency recommending the treatment be given orphan drug status in…
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…