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...
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...
Rare Pediatric Disease Designation is granted by the FDA for serious or life-threatening diseases which primarily affect individuals less than 18 years of age and fewer than 200,000 people in the United States. If a New Drug Application for WVE-N531 is approved by the FDA, Wave would be el...
FDA grants rare pediatric disease designation for Leber congenital amaurosis gene therapyYoung, AlexOcular Surgery News
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...
Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company) today announced the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation to TNX-2900* (intranasal potentiated oxytocin), a proprie...
(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 Descartes-08 for the treatment of juveni...
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. ...
Akari Therapeutics, Plc recently announced the US FDA has granted the Rare Pediatric Disease Designation to nomacopan for the treatment of pediatric
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