ImmuneSensor Therapeutics receives orphan drug and rare paediatric disease designations from the US FDA for cGAS inhibitor drug candidate, IMSB301, for the treatment of Aicardi Goutières syndrome

20 November 2024 - ImmuneSensor Therapeutics announced that the US FDA has granted both orphan drug designation and rare paediatric disease ...

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Alkeus Pharmaceuticals receives FDA rare paediatric disease and fast track designations for gildeuretinol as a treatment for Stargardt disease

18 November 2024 - Alkeus Pharmaceuticals today announced that gildeuretinol (ALK-001), an investigational oral therapy, has received rare paediatric disease and ...

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AlveoGene receives rare paediatric disease designation from FDA for AVG-002, its novel, inhaled gene therapy for lethal neonatal surfactant protein B deficiency

15 November 2024 -  AlveoGene announces that it has been granted a rare paediatric disease designation by the US FDA ...

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Actuate announces FDA rare paediatric disease designation granted to elraglusib for treatment of Ewing sarcoma

12 November 2024 - On-going enrolment in Phase 1/2 trial of elraglusib in relapsed/refractory Ewing sarcoma with topline Phase 1 data ...

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Cumberland Pharmaceuticals receives FDA orphan drug and rare paediatric disease designations for new treatment of Duchenne muscular dystrophy

6 November 2024 -  Cumberland Pharmaceuticals announced today that the US FDA granted orphan drug designation and rare paediatric disease ...

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AskBio receives FDA rare paediatric disease and orphan drug designations for AB-1003 for the treatment of limb girdle muscular dystrophy type 2I/R9

7 November 2024 - First patient dosed in Phase 1/Phase 2 LION-CS101 trial of AB-1003 in August 2023, with enrolment ...

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Dupixent (dupilumab) approved in the European Union as the first and only medicine for young children with eosinophilic oesophagitis

6 November 2024 - Approval based on Phase 3 data showing significantly more children aged 1 to 11 years on ...

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NS parents dismayed by lack of access to new RSV vaccines for infants

3 November 2024 - Officials say there is no universally available, publicly funded RSV vaccine program for newborns in Nova Scotia. ...

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FDA grants rare paediatric disease designation to Omeros’ MASP-3 inhibitor zaltenibart for treatment of C3 glomerulopathy

24 October 2024 - Phase 3 studies on the horizon in both PNH and C3G. ...

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Papillon Therapeutics receives rare paediatric disease designation from the US FDA for PPL-002 for the treatment of Danon disease

21 October 2024 - Papillon Therapeutics today announced that the US FDA has granted rare paediatric disease designation to Papillon’s PPL-002, ...

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Avadel Pharmaceuticals announces FDA approval of Lumryz (sodium oxybate) extended release oral suspension for the treatment of cataplexy or excessive daytime sleepiness in patients 7 years of age and older with narcolepsy

17 October 2024 - Granted orphan drug exclusivity in paediatric narcolepsy patients 7 years and older through 16 October 2031. ...

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Sellas announces US FDA rare paediatric disease designation granted to galinpepimut-S for the treatment of paediatric acute myeloid leukaemia

15 October 2024 - Galinpepimut-S currently investigated in Phase 3 REGAL trial in adult AML patients – interim analysis anticipated in ...

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FDA grants rare paediatric disease designation to MDL-101 for the treatment of congenital muscular dystrophy type 1a

30 September 2024 - Modalis Therapeutics today announced that the US FDA has granted rare paediatric disease designation to MDL-101, a ...

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Enterprise Therapeutics granted rare paediatric disease designation in the US for novel cystic fibrosis investigational therapy ETD001

26 September 2024 - ETD001 is a novel, first in class blocker of the epithelial sodium channel aimed at treating people ...

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Lantern Pharma announces three US FDA rare paediatric disease designations granted to LP-184 in multiple ultra rare children’s cancers

23 September 2024 - Lantern Pharma announced today that the company has been granted three rare paediatric disease designations by the ...

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