'A child's health should not be a postcode lottery': anger of cystic fibrosis sufferers forced to pay £20,000 for drug abroad

17 September 2019 - Patients have formed a buyers' club to source a version of Orkambi after the drug's manufacturer and ...

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News NHS England explores funding options for cystic fibrosis drug

17 September 2019 - NHS England is “committed to exploring and developing alternative routes” to enable people with cystic fibrosis ...

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Vertex announces new access agreement with Scottish Government for Orkambi (lumacaftor/ivacaftor) and Symkevi (tezacaftor/ivacaftor)

12 September 2019 - Eligible patients in Scotland will immediately have access to Orkambi (lumacaftor/ivacaftor) and Symkevi (tezacaftor/ivacaftor) in combination with ...

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Batten disease treatment: parents win battle ahead of court date

11 September 2019 - Families of children with a rare degenerative disease have won their fight for funding for NHS ...

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Nucala is the first biologic approved in the US for six to 11-year-old children with severe eosinophilic asthma

12 September 2019 - GlaxoSmithKline today announced that the US FDA has approved Nucala (mepolizumab) for use in children as young ...

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Children with rare inherited condition to benefit from drug through managed access agreement

11 September 2019 - It is estimated that in the UK there are around 30 to 50 children living with the ...

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Kiwi toddler fights spinal muscular atrophy in Australia after PHARMAC stall access to life-saving drug

7 September 2019 - For the first time in her life, two-year-old Charlotte Bond is showing signs that one day ...

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GeneTx and Ultragenyx announce orphan drug designation and rare paediatric disease designation for GTX-102

3 September 2019 - GeneTx Biotherapeutics and Ultragenyx today announced that the U.S. FDA has granted orphan drug designation and rare ...

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Russia approves Spinraza to treat all SMA patients

29 August 2019 - Russia has become the first of the 15 former Soviet republics to approve Biogen’s Spinraza (nusinersen) ...

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Promoting paediatric drug research and labelling — outcomes of legislation

28 August 2019 - Congress has modified regulations of the FDA to promote research assessing the efficacy and safety of drugs ...

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Free flu jabs for kids on NIP move is welcomed by state Health minister Steven Miles

28 August 2019 - Australian children aged six months to five will be given free flu vaccines in 2020 if Health ...

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Family’s joy at access to drug for son

27 August 2019 - A Bunbury mother says she has more hope for her son’s future following a recommendation to ...

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‘Life saving’ news for Gold Coast family after little Beau allowed to take drug Orkambi to slow cystic fibrosis diagnosis

23 August 2019 - When this Gold Coast girl blew out candles for her 10th birthday yesterday, she did not wish ...

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Fiasp approved in Europe for the management of diabetes in adolescents and children aged one year and above

22 August 2019 - Novo Nordisk today announced that the European Commission has granted an extension of the indication for ...

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Teva launches generic version of EpiPen for young children

20 August 2019 - Teva Pharmaceutical Industries on Tuesday made its generic version of Mylan’s EpiPen for young children available in ...

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