17 October 2023 - Approximately 330 children with cystic fibrosis ages 2-5 years are now eligible for a medicine that ...
17 October 2023 - Rare paediatric disease designation underscores critical need for novel therapies to address IFALD. ...
6 October 2023 - Expanded indication for Zoryve provides new, steroid free topical for children 6 to 11 with plaque ...
27 September 2023 - Shionogi announced the US FDA has granted Tetra Therapeutics, a Shionogi Group Company, rare paediatric disease designation ...
26 September 2023 - Today, the FDA approved bosutinib (Bosulif, Pfizer) for paediatric patients 1 year of age and older ...
26 September 2023 - If approved, Dupixent would be the first and only treatment in the US indicated for children aged ...
12 September 2023 - Final results from XTEND-Kids study were submitted for review, potentially expanding on the interim data included in ...
15 September 2023 - BioMarin today announced that the EMA's CHMP has adopted a positive opinion recommending marketing authorisation to expand ...
15 September 2023 - If approved, more than 1,200 children would be newly eligible for a medicine that could treat ...
7 September 2023 - Milestone signifies on-going effort to help prepare for outbreaks of Zaire ebolavirus. ...
5 September 2023 - Families calling for greater access to a medication that helps manage a severe form of epilepsy ...
29 August 2023 - Approval is based on interim data from ongoing RAINBOWFISH trial showing majority of Evrysdi-treated babies were able ...
29 August 2023 - Bloomsbury Genetic Therapies Limited announced today that the US FDA has granted rare paediatric disease designation for ...
14 August 2023 - LCTG-001 on course to be the first FDA approved biologic from human milk. ...
14 August 2023 - Emma Albee knows the experimental drug she takes is not a cure. It won’t allow her ...