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Precision BioSciences Receives FDA Rare Pediatric Disease Designation for PBGENE-DMD for the Treatment of Duchenne Muscular Dystrophy
Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® ...
Quoin Pharmaceuticals Announces FDA Grants Rare Pediatric Disease Designation for QRX003 in Netherton Syndrome
Quoin Pharmaceuticals Ltd. (NASDAQ: QNRX) (“Quoin” or the “Company”), a late clinical-stage specialty pharmaceutical company ...
Minovia Therapeutics Announces FDA Fast Track and Rare Pediatric Disease Designations for MNV-201 in Pearson Syndrome
Regulatory Milestone Underscores Urgency and Promise of First-in-Class Mitochondrial Cell TherapyHaifa, ISRAEL, June 30, 2025 (GLOBE NEWSWIRE) -- Minovia Therapeutics Ltd. (“Minovia” or the “Company”) ...
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FDA grants rare pediatric disease designation for Netherton syndrome drug
The FDA’s Rare Pediatric Disease Designation program aims to encourage development of therapies for serious diseases primarily affecting individuals under 18 years of age. If a New Drug Application ...
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FDA grants rare pediatric disease designation for Netherton syndrome drug
ASHBURN, Va. - The U.S. Food and Drug Administration has granted Rare Pediatric Disease Designation for QRX003, a drug being developed by Quoin Pharmaceuticals Ltd. (NASDAQ:QNRX) to treat ...
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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).
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Precision BioSciences Receives FDA Rare Pediatric Disease Designation for PBGENE-DMD for the Treatment of Duchenne Muscular Dystrophy - Morningstar
The FDA grants Rare Pediatric Disease Designation to therapies targeting serious and life-threatening diseases that affect fewer than 200,000 people in the U.S. and primarily affect individuals ...
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Quoin Pharmaceuticals Announces FDA Grants Rare Pediatric Disease Designation for QRX003 in Netherton Syndrome - Morningstar
“We are very pleased to announce the receipt of Rare Pediatric Disease Designation for QRX003 for Netherton Syndrome, a severe and underserved genetic disease,” said Dr. Michael Myers, Chief ...
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Precision BioSciences Gets FDA's Rare Pediatric Disease Designation For PBGENE-DMD - Nasdaq
(RTTNews) - Precision BioSciences, Inc. (DTIL) announced Wednesday that the U.S. Food and Drug Administration or FDA has granted Rare Pediatric Disease Designation for PBGENE-DMD, the company's ...
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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 ...
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VG801 gene therapy in Stargardt disease receives rare pediatric disease designation - Healio
“The FDA rare pediatric disease designation for VG801 not only highlights the strength of our scientific approach but also reinforces our focus on accelerating the development and delivery of ...
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Quoin Pharmaceuticals receives FDA rare pediatric disease designation for QRX003 - Investing.com
Quoin Pharmaceuticals Ltd. (NASDAQ:QNRX) announced Tuesday that the U.S. Food and Drug Administration has granted Rare Pediatric Disease Designation to its lead asset, QRX003, for the treatment of ...