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The US FDA Grants Both Orphan Drug and Rare Pediatric Disease Designations to NEU-001 of Neurenati for the Treatment of Hirschsprung Disease

Christian Girard

Corporate Advisor | Alternative Funding | Orphan Drugs | Rare Pediatric Disease Designation | Priority Review Voucher

发布日期: 2025年3月5日

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The PRV Chronicle

785 位关注者

Christian Girard的更多文章

Innorna Announces FDA Rare Pediatric Disease Designation Granted to IN013 for Treatment of Wilson Disease

Zevra Therapeutics Enters Agreement to Sell its Rare Pediatric Disease Priority Review Voucher for $150 Million

Neuren receives Rare Pediatric Disease Designations from FDA

Neuren Pharmaceuticals received Priority Review Voucher sale proceeds from partner Acadia

Ensoma Receives Rare Pediatric Disease and Orphan Drug Designations for EN-374 for Treatment of X-linked Chronic Granulomatous Disease (X-CGD)

SpringWorks granted rare pediatric disease priority review voucher by the FDA

FDA grants CORDStrom a Rare Pediatric Disease Designation (RPDD) and Orphan Drug Designation (ODD) for treatment of epidermolysis bullosa (EB)

Arbor Biotechnologies Announces FDA Orphan Drug and Rare Pediatric Disease Designations Granted to ABO-101 for the Treatment of Primary Hyperoxaluria

SEED Receives FDA Rare Pediatric Disease and Orphan Drug Designations for Lead Oncology Asset RBM39 Degrader

FDA Grants Rare Pediatric Disease Designation to NS-051/NCNP-04 for the Treatment of Duchenne Muscular Dystrophy