News

Research Moves Slowly. Rare Diseases Don't—So Patients Aren't Waiting
Patients with rare diseases are increasingly working to find and fund their own cures. But should they have to?
TMCnet2d
Belite Bio Announces Completion of Enrollment in the Pivotal Global Phase 3 PHOENIX Trial Evaluating Oral Tinlarebant in Geographic Atrophy
The PHOENIX study is a 24-month, randomized, double-masked, placebo-controlled, multicenter, pivotal Phase 3 trial. The study is ongoing across sites in the United States, the United Kingdom, France, ...
Minovia Gets FDA Designations Supporting Development of Pearson Syndrome Treatment
Minovia Therapeutics said Monday that the Food and Drug Administration granted fast-track and rare-pediatric-disease designations to its lead investigational compound. The drug, MNV-201, is in Phase 2 ...
InvestorsHub on MSN8d
Precision BioSciences Shares Surge Following FDA Rare Pediatric Disease Designation
Shares of Precision BioSciences, Inc. (NASDAQ:DTIL) jumped 17% on Wednesday after the company announced that its gene therapy ...
Precision BioSciences gets FDA rare disease status for DMD treatment, stock jumps 10%
Precision BioSciences (NASDAQ:DTIL)  announced Wednesday that its experimental therapy PBGENE-DMD has received Rare Pediatric ...
Contract Pharma8d
Precision BioSciences Gains FDA Rare Pediatric Disease Designation for PBGENE-DMD
Precision BioSciences, Inc. received a Rare Pediatric Disease Designation from the U.S. Food and Drug Administration (FDA) for PBGENE-DMD to treat Duchenne muscular dystrophy (DMD).
TMCnet9d
Quoin Pharmaceuticals Announces FDA Grants Rare Pediatric Disease Designation for QRX003 in Netherton Syndrome
The designation reinforces the potential of QRX003 as a therapeutic candidate for a profoundly underserved pediatric population. It follows earlier regulatory recognition by the European Medicines ...
Yahoo Finance1mon
Ocugen Announces Rare Pediatric Disease Designation Granted for OCU410ST—Modifier Gene Therapy for the Treatment of Stargardt Disease - Yahoo Finance
With this designation for OCU410ST, Ocugen may be awarded a Priority Review Voucher (PRV), if the PRV program is reauthorized by the U.S. Congress. The PRV program is designed to incentivize drug ...
Healio1mon
FDA grants rare pediatric disease designation for Stargardt disease gene therapy - Healio
The FDA granted rare pediatric disease designation for OCU410ST, a gene therapy designed to treat ABCA4-associated retinopathies including Stargardt disease, retinitis pigmentosa 19 and cone-rod ...
PharmiWeb1mon
Relief Therapeutics Receives FDA Rare Pediatric Disease Designation for RLF-TD011
GENEVA (May 22, 2025) – RELIEF THERAPEUTICS Holding SA (SIX: RLF, OTCQB: RLFTF, RLFTY) (Relief or the Company), a biopharmaceutical company committed to delivering innovative treatment options for ...
Cure Today3mon
FDA Grants Rare Pediatric Designation to Treatment With NEO100 in Brain Cancer
Key Takeaways The FDA's designation for NEO100 highlights the urgent need for treatments for pediatric-type diffuse high-grade gliomas, a rare and aggressive brain cancer. NeOnc Technologies may ...
Morningstar3mon
NeOnc Technologies Holdings, Inc. Receives Rare Pediatric Disease Designation for NEO100 in Treatment of Pediatric-Type Diffuse High-Grade Gliomas | Morningstar
“Receiving the Rare Pediatric Disease Designation for NEO100 is a crucial step forward in our mission to develop effective treatments for children facing diffuse high-grade gliomas,” said Dr ...