Newron receives FDA rare paediatric disease designation for sarizotan for the treatment of Rett syndrome

19 November 2019 - Newron announced today that the U.S. FDA has granted the rare paediatric disease designation for sarizotan, ...

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Anavex Life Sciences receives rare paediatric disease designation from FDA for Anavex 2-73 (blarcamesine) for the treatment of Rett syndrome

14 November 2019 - Anavex Life Sciences today announced that it received the rare paediatric disease designation from the U.S. FDA ...

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Talking with Tama, a sick toddler who can't access the drug he needs

5 November 2019 - "Hi!" a bright young voice says down the phone. ...

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Tama is deteriorating fast. But the drug that can help him is too expensive.

31 October 2019 - Tama Hubbard has good days and bad days, just like any other 3-year-old child. ...

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'Why won’t they help him?': 'Broken' mother pleads for PHARMAC funding for toddler's rare disorder

1 November 2019 - The mother of a young boy is pleading with PHARMAC to fund a drug that could ...

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Intravenous Benlysta is the first biologic treatment to be approved for children with lupus in Europe

29 October 2019 - GlaxoSmithKline today announced that the European Commission has adopted a decision to extend to children five years ...

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FDA approves Botox (onabotulinumtoxinA) for paediatric patients with lower limb spasticity, excluding spasticity caused by cerebral palsy

24 October 2019 - Approval marks 11th Botox therapeutic indication and comes 30 years after the first indications were approved. ...

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Cystic fibrosis medicines listings on the Pharmaceutical Benefits Scheme

21 October 2019 - Minister Hunt talks at the Great Strides Fun Run about the listing of 2 new medicines, ...

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Vertex announces reimbursement of cystic fibrosis medicines Symdeko (tezacaftor/ivacaftor and ivacaftor) for eligible patients ages 12 and older, and Orkambi (lumacaftor/ivacaftor) in children ages 2 to 5, with certain CFTR mutations in Australia

19 October 2019 - Vertex Pharmaceuticals today announced that Symdeko (tezacaftor/ivacaftor and ivacaftor) is reimbursed in Australia for people with cystic ...

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ProQR receives rare paediatric disease designation from FDA for sepofarsen for the treatment of LCA10

15 October 2019 - ProQR Therapeutics today announced that it received rare paediatric disease designation from the U.S. FDA for ...

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Faced with a drug shortfall, doctors scramble to treat children with cancer

14 October 2019 - A critical chemotherapy medication is in short supply, and physicians say there is no appropriate substitute. ...

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'I'm not ready to bring her back' - NZ not safe for three-year-old with motor neuron disease, mum says

10 October 2019 - Three-year-old New Zealander Charlotte Bond is showing signs she may walk again on her own with ...

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Prescription opioid dispensing in Australian children and adolescents: a national population-based study

8 October 2019 - There are few population-based studies of paediatric opioid use.  ...

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Janssen submits application to U.S. FDA seeking approval of Stelera (ustekinumab) for the treatment of paediatric patients with moderate to severe plaque psoriasis

7 October 2019 - The Janssen Pharmaceutical Companies of Johnson & Johnson today announced the submission of a supplemental biologics license ...

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Novartis Entresto receives FDA approval for paediatric heart failure, helping to address critical unmet need for treatment options

1 October 2019 - Entresto is now approved for the treatment of paediatric patients aged 1 year and older with symptomatic ...

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