The FDA has granted a rare pediatric disease designation to GPS for the treatment of pediatric patients with AML.1 "GPS has already demonstrated promise in clinical settings for AML, and we believe its potential could extend to pediatric patients,” said Angelos Stergiou, MD, ScD hc, president...
“This designation from FDA underscores that significant unmet needs remain in DMD, and it also supports Wave’s innovative and purposeful approach to drug development in the rare disease space,” said Anne-Marie Li-Kwai-Cheung, MChem, MTOPRA, RAPS, Chief Development Officer at Wave Life Science...
FDA grants rare pediatric disease designation for Leber congenital amaurosis gene therapyYoung, AlexOcular Surgery News
The FDA granted rare pediatric disease designation for BPM31510T for the treatment of epidermolysis bullosa.Image: Adobe Stock. “We are honored to offer hope for children who suffer from debilitating wounds and pain,”Vijay Modur, MD, PhD,chief medical officer of BPGbio...
The FDA has granted the novel GSK-3β inhibitor elraglusib rare pediatric disease designation for the potential treatment of patients with Ewing sarcoma.1 “Receiving rare pediatric disease designation from the FDA underscores the urgent need for new treatment options for patients with Ewing sarcoma...
安济盛生物近日宣布获得美国FDA授予其在研新药AGA2115罕见儿科疾病认定(Rare Pediatric Disease Designation, RPDD),用于治疗成骨不全症(Osteogenesis Imperfecta, OI)。 成骨不全症是一种严重危害生命的遗传性疾病。作为一种罕见的儿科疾病,OI主要发生于新生儿和儿童,表现为频发性骨折。严重的OI病例可表现为多处骨折,...
US FDA grants Rare Pediatric Disease Designation to Axovant’s AAV9-based gene therapy, AXO-AAV-GM1 for GM1 gangliosidosis
QSAM Biosciences Inc. recently announced the US FDA has granted Rare Pediatric Disease Designation to QSAM’s clinical-stage drug candidate, CycloSam, for the treatment osteosarcoma, a devastating form of bone cancer that afflicts mostly children and young adults. ...
(RTTNews) - Cartesian Therapeutics, Inc. (RNAC), a clinical-stage biotechnology company pioneering mRNA cell therapy for autoimmune diseases, announced Monday that the U.S. Food and Drug Administration has granted Rare Pediatric Disease Designation to De
Akari Therapeutics, Plc recently announced the US FDA has granted the Rare Pediatric Disease Designation to nomacopan for the treatment of pediatric