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…
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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…
Strenuous, intense exercise done frequently is an environmental risk factor for amyotrophic lateral sclerosis in people with ALS risk genes, particularly those who carry a faulty C9ORF72 gene, a study concluded. Future research is needed to better understand and identify people at disease risk due to such exercise,…
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…
Alector and GlaxoSmithKline (GSK) have established a partnership to develop two investigational antibodies — called AL001 and AL101 — to boost the production of the progranulin protein as a potential treatment for neurodegenerative diseases, including amyotrophic lateral sclerosis (ALS). Low levels of progranulin, known as PGRN, which is a…
The first part of Biogen’s early access program for tofersen is now open to patients with the most rapidly progressing cases of familial amyotrophic lateral sclerosis (ALS), the company announced. These patients, with familial ALS caused by mutations in the SOD1 gene, will be the first to…
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…
At its first virtual investor event, biotech company Centogene set a bold mission: to cure 100 rare diseases within the next decade. A leader in the field of genetic diagnostics, Centogene used the June 22 event to present its strategic priorities, outlining its plans to speed the discovery…
Exservan, the first oral film formulation of riluzole for treating amyotrophic lateral sclerosis (ALS), has been launched in the United States, its developer, Mitsubishi Tanabe Pharma America (MTPA), has announced. Rapidly dissolved when placed on top of the tongue, Exservan — specifically developed to help meet the needs…
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…
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