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…
On one of my recent daily walks, I listened to an “Office Ladies” podcast in which Jenna Fischer shared her ongoing struggle with anxiety. She used the analogy of a backpack to describe the burden she lives with. Some days it weighs her down, while other days she…
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…
Everyone likes being noticed — for the right things, of course. I like it when others recognize my unique talents and skills or a job well done. I don’t like being noticed because I have to use a rollator to help me walk. Or, because I move more slowly than…
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…
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