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RSS FeedPosted by Michael Wonder on 10 Sep 2024
Cartesian Therapeutics receives FDA rare paediatric disease designation for Descartes-08 for the treatment of juvenile dermatomyositis
9 September 2024 - Cartesian Therapeutics today announced that the US FDA has granted rare paediatric disease designation to Descartes-08 ...
Posted by Michael Wonder on 08 Sep 2024
YolTech Therapeutics receives US FDA rare paediatric disease designation for YOLT-203 in treating primary hyperoxaluria type 1
4 September 2024 - YolTech Therapeutics today announced that the US FDA has granted rare paediatric disease designation to YOLT-203 for ...
Posted by Michael Wonder on 02 Sep 2024
CAMP4 Therapeutics secures rare paediatric disease designation for CMP-CPS-001 for the treatment of urea cycle disorders
27 August 2024 - CAMP4 Therapeutics today announced the US FDA has granted rare paediatric disease designation to the Company’s lead ...
Posted by Michael Wonder on 27 Aug 2024
Rhythm Pharmaceuticals announces FDA acceptance for priority review of supplemental new drug application for Imcivree (setmelanotide) in patients as young as 2 years old
26 August 2024 - FDA sets PDUFA goal date of 26 December 2024. ...
Posted by Michael Wonder on 22 Aug 2024
Opus Genetics receives rare paediatric disease designation from the US FDA for ocular gene therapy OPGx-LCA5 to treat rare inherited retinal disease LCA5
20 August 2024 - Opus Genetics today announced the US FDA has granted rare paediatric disease designation for its ocular gene ...
Posted by Michael Wonder on 13 Aug 2024
Wave Life Sciences receives FDA rare paediatric disease designation for WVE-N531 for the treatment of Duchenne muscular dystrophy
12 August 2024 - Wave Life Sciences today announced that the US FDA has granted rare paediatric disease designation to WVE-N531 ...
Posted by Michael Wonder on 12 Aug 2024
Actio Biosciences receives orphan drug and rare paediatric disease designations for the treatment of Charcot-Marie-Tooth disease 2C
8 August 2024 - Actio Biosciences today announced the US FDA has granted both orphan drug designation and rare paediatric disease ...
Posted by Michael Wonder on 11 Aug 2024
Satellos receives rare paediatric disease designation from the US FDA for SAT-3247 for the treatment of Duchenne muscular dystrophy
8 August 2024 - Satellos Bioscience announced today that the US FDA has granted rare paediatric disease designation to SAT-3247 ...
Posted by Michael Wonder on 07 Aug 2024
Meta Pharmaceuticals announces FDA grants rare paediatric disease designation to META-001-PH for the treatment of primary hyperoxaluria
5 August 2024 - META Pharmaceuticals Inc. announced that the US FDA has granted rare paediatric disease designation to its investigational ...
Posted by Michael Wonder on 05 Aug 2024
FDA submission to expand minimum age for Xenoview
1 August 2024 - Polarean Imaging announces that it has submitted a new drug application supplement to the US FDA to ...
Posted by Michael Wonder on 03 Aug 2024
Drugs in rare paediatric disease priority review voucher program have similar revenue to brand drugs
2 August 2024 - Drugs issued rare paediatric disease priority review vouchers that were redeemed had revenues that were similar ...
Posted by Michael Wonder on 26 Jul 2024
US FDA approves BioMarin's Brineura (cerliponase alfa) for children under 3 years with CLN2 disease
24 July 2024 - Now approved for children of all ages with CLN2 Batten disease, regardless of whether they yet ...
Posted by Michael Wonder on 25 Jul 2024
ConSynance Therapeutics receives FDA rare paediatric disease designation for CSTI-500 in Prader-Willi syndrome
24 July 2024 - ConSynance Therapeutics today announced that the US FDA has granted rare paediatric disease designation to its ...
Posted by Michael Wonder on 24 Jul 2024
FDA accepts Mesoblast's biologics license application for Ryoncil in children with steroid-refractory acute graft versus host disease
23 July 2024 - Mesoblast announced that the US FDA has accepted its biologics license application resubmission for Ryoncil (remestemcel-L) ...
Posted by Michael Wonder on 20 Jul 2024
Neuren receives rare paediatric disease designation from FDA
19 July 2024 - Neuren Pharmaceuticals has received rare paediatric disease designation from the US FDA for NNZ-2591 in Phelan-McDermid ...