The FDA grants rare pediatric disease designation for serious or life-threatening diseases that affect fewer than 200,000 people in the United States when those people are primarily younger than 18 ...

The FDA granted galinpepimut-S a rare pediatric disease designation for the treatment of pediatric patients with acute ...

SELLAS Life Sciences Group, Inc. (SLS), a late-stage clinical biopharmaceutical company, Tuesday announced that the U.S. Food and ...

has been granted ‘rare pediatric disease designation’ (RPD) in the US by the Food and Drug Administration (FDA).1 ETD001 is a low molecular weight compound with first-in-class potential ...

BOSTON–(BUSINESS WIRE)–#bpgbio—BPGbio, Inc., a leading biology-first, AI-powered, clinical stage biopharma focused on mitochondrial biology and protein homeostasis, today announced that the U.S. Food ...

has granted Rare Pediatric Disease (RPD) designation to MDL-101, a novel precision medicine being developed for the treatment of congenital muscular dystrophy type 1a (LAMA2-CMD). The FDA grants ...

If the rare pediatric disease PRV program is not reauthorized, eligibility to receive a voucher will be limited to products that receive rare pediatric disease designation by December 20, 2024, and ...