News

ABO-202 Earns EMA’s Orphan Drug Status for Infantile Batten Disease

ABO-202, Abeona Therapeutics‘ investigational candidate for the treatment of infantile Batten disease has received orphan drug status by the European Medicines Agency. This new status adds to the previously granted orphan drug and rare pediatric disease designation by the U.S. Food and Drug Administration. These designations are…

Genome Sequencing Accurately, Rapidly Diagnosed CLN8 Variants in Boy, 8

Chinese researchers used an advanced genetic sequencing technique to identify two new pathogenic variants of the CLN8 gene in a boy with suspected neuronal ceroid lipofuscinosis (NCL), or Batten disease. Molecular diagnosis was delivered within three weeks, demonstrating the advantages of using genome sequencing for providing fast and…

NIH Agency Pioneers Collaborative Research into Rare Diseases

A little-known government entity within the National Institutes of Health (NIH) is helping to lead U.S. efforts to speed up the development of therapies for some 7,000 rare diseases. The Office of Rare Diseases Research (ORDR), headquartered in Bethesda, Maryland, was established in 1993 within the NIH Office of the…