U.S. Food and Drug Administration approves Lampit (nifurtimox) for the treatment of Chagas disease in children

7 August 2020 - Only Chagas disease treatment approved in U.S. for use in children from birth to less than 18 ...

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US FDA grants rare paediatric disease designation to paxalisib for DIPG

7 August 2020 - Kazia Therapeutics is pleased to announce that the United States FDA has awarded rare paediatric disease designation ...

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Cerecor receives rare paediatric disease designation for CERC-006 in lymphatic malformations

4 August 2020 - Cerecor today announced that the U.S. FDA granted rare paediatric disease designation to CERC-006, a dual inhibitor ...

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Pacira BioSciences announces FDA acceptance of sNDA for Exparel use in paediatric patients

4 August 2020 - sNDA submission based on positive Phase 3 data supporting expansion of the Exparel label to include use ...

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Novartis Cosentyx receives EU approval for first-line systemic treatment in paediatric psoriasis

3 August 2020 - EU approval is based on two Phase III studies showing Cosentyx provides fast and strong skin clearance, ...

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U.S. Food and Drug Administration approves Stelara (ustekinumab) for treatment of paediatric patients with moderate to severe plaque psoriasis

30 July 2020 - Stelara is the first and only biologic to target interleukin (IL)-12 and IL-23 approved for paediatric psoriasis ...

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Marinus Pharmaceuticals receives rare paediatric disease designation from FDA for ganaxolone for the treatment of CDKL5 deficiency disorder

30 July 2020 - Marinus Pharmaceuticals announced today that the U.S. FDA has granted rare paediatric disease designation for the ...

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Health Canada Approves Brivlera (brivaracetam) to treat partial-onset seizures in paediatric epilepsy patients

29 July 2020 - Health Canada's approval of Brivlera provides paediatric epilepsy patients with a treatment option which can be initiated ...

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Protara Therapeutics receives rare paediatric disease designation for TARA-002 for the treatment of lymphatic malformations

28 July 2020 - Protara Therapeutics today announced that the U.S. FDA has granted rare paediatric disease designation for TARA-002 for ...

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HemoShear Therapeutics receives FDA fast track and rare paediatric disease designations for HST5040 to treat methylmalonic acidaemia and propionic acidaemia

28 July 2020 -  HemoShear Therapeutics has received fast track and rare paediatric disease designations from the U.S. FDA for ...

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Momenta Pharmaceuticals announces FDA rare paediatric disease designation for nipocalimab in HDFN

28 July 2020 - Momenta Pharmaceuticals today announced that its novel drug candidate, nipocalimab, has received rare paediatric disease designation ...

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'I need something to move forward,' says Edmonton mom waiting for approval for son's life saving treatment

24 July 2020 - The family of a little boy in need of a $3 million treatment says they have ...

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Mother of teen with cystic fibrosis pleads for PHARMAC to fund new medication

26 July 2020 - Seventeen-year-old Rotorua teenager Osiris Daniels has been living in isolation his entire life, a necessary requirement ...

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BioMarin submits marketing authorisation application to European Medicines Agency for vosoritide to treat children with achondroplasia

23 July 2020 - If approved, vosoritide would be the first medicine to treat Achondroplasia in EU. ...

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'Severe atopic dermatitis drug too expensive for adolescents'

21 July 2020 - The drug regulator expanded approval for Dupixent (dupilumab), a treatment for severe atopic dermatitis, from adults ...

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