20 November 2024 - ImmuneSensor Therapeutics announced that the US FDA has granted both orphan drug designation and rare paediatric disease ...
18 November 2024 - Alkeus Pharmaceuticals today announced that gildeuretinol (ALK-001), an investigational oral therapy, has received rare paediatric disease and ...
15 November 2024 - AlveoGene announces that it has been granted a rare paediatric disease designation by the US FDA ...
12 November 2024 - On-going enrolment in Phase 1/2 trial of elraglusib in relapsed/refractory Ewing sarcoma with topline Phase 1 data ...
6 November 2024 - Cumberland Pharmaceuticals announced today that the US FDA granted orphan drug designation and rare paediatric disease ...
7 November 2024 - First patient dosed in Phase 1/Phase 2 LION-CS101 trial of AB-1003 in August 2023, with enrolment ...
6 November 2024 - Approval based on Phase 3 data showing significantly more children aged 1 to 11 years on ...
3 November 2024 - Officials say there is no universally available, publicly funded RSV vaccine program for newborns in Nova Scotia. ...
24 October 2024 - Phase 3 studies on the horizon in both PNH and C3G. ...
21 October 2024 - Papillon Therapeutics today announced that the US FDA has granted rare paediatric disease designation to Papillon’s PPL-002, ...
17 October 2024 - Granted orphan drug exclusivity in paediatric narcolepsy patients 7 years and older through 16 October 2031. ...
15 October 2024 - Galinpepimut-S currently investigated in Phase 3 REGAL trial in adult AML patients – interim analysis anticipated in ...
30 September 2024 - Modalis Therapeutics today announced that the US FDA has granted rare paediatric disease designation to MDL-101, a ...
26 September 2024 - ETD001 is a novel, first in class blocker of the epithelial sodium channel aimed at treating people ...
23 September 2024 - Lantern Pharma announced today that the company has been granted three rare paediatric disease designations by the ...