News

AcuraStem has been awarded a $1 million grant from the U.S. Department of Defense (DOD) to advance AS-202, a potentially disease-modifying therapy for sporadic amyotrophic lateral sclerosis (ALS), according to a press release. The candidate therapy is designed to increase the number of lysosomes — cell compartments…

Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European action plan for rare diseases to ensure that none of the 30 million people in Europe living with rare diseases are left behind by the start of a new decade. As part of…

PharmaTher has filed an application with the U.S. Food and Drug Administration (FDA) requesting that ketamine be designated an orphan drug as a potential treatment of amyotrophic lateral sclerosis (ALS). Orphan drug status is granted by the FDA to encourage the development of therapies for conditions that affect fewer…

The European Medicines Agency (EMA) has granted orphan drug designation to Seelos Therapeutics‘ investigational therapy SLS-005 for the treatment of amyotrophic lateral sclerosis (ALS), the company announced in a press release. Orphan drug status in Europe is given to medicines with the potential to be safe and…

Ahead of this year’s Rare Disease Week on Capitol Hill, held virtually July 14–22, the EveryLife Foundation will award grants to top advocates of rare disease organizations who participate in the week’s pre-events. The top 50 point-earners will be eligible to win $1,000 to $5,000 in grants, totaling…

AB Science will suspend recruitment and treatment in all studies involving masitinib, a tyrosine kinase inhibitor being investigated for amyotrophic lateral sclerosis (ALS) and several other conditions. The voluntary suspension follows the identification of a potential risk of ischemic heart disease — a condition of recurring chest…

Today, the National ALS Registry is joining the Major League Baseball to celebrate Lou Gehrig’s life and legacy and to raise greater awareness of amyotrophic lateral sclerosis (ALS). This year’s inaugural “Lou Gehrig Day” on June 2 honors the famous New York Yankees baseball player whose career…

People living with amyotrophic lateral sclerosis (ALS) recently shared their views on the importance of new therapies and incremental gains, and the risks they are willing to take with U.S. Food and Drug Administration (FDA) and industry officials in a virtual meeting hosted by The ALS Association. The…

Researchers at the University of Texas at Dallas (UT Dallas) have received an award from the U.S. Army Medical Research Acquisition Activity to advance their preclinical research of an innovative CRISPR-based gene editing approach to treat one of the most common causes of familial amyotrophic lateral sclerosis (ALS). The two-year…

At face value, the purpose of the National Amyotrophic Lateral Sclerosis (ALS) Registry seems quite straightforward: collecting data on the U.S. population with ALS. But there’s a lot more to it. The registry, funded by the…