This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, the potential significance and implications of receiving the Rare Pediatric Disease Designation from the FDA; the potentially registra...
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...
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...
“Receiving rare pediatric disease designation from the FDA underscores the urgent need for new treatment options for patients with Ewing sarcoma and recognizes elraglusib’s transformative potential,” Daniel Schmitt, president and chief executive officer of Actuate Therapeutics, stated in a news relea...
Join the 14th World Congress on Rare Diseases and Orphan Drugs, June 2-3, 2025, in Amsterdam. Explore breakthroughs in research, treatment, and innovation to transform rare disease care globally.
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...
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Akari Therapeutics, Plc recently announced the US FDA has granted the Rare Pediatric Disease Designation to nomacopan for the treatment of pediatric
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. ...