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RSS FeedPosted by Michael Wonder on 20 Nov 2019
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, ...
Posted by Michael Wonder on 14 Nov 2019
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 ...
Posted by Michael Wonder on 05 Nov 2019
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. ...
Posted by Michael Wonder on 03 Nov 2019
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. ...
Posted by Michael Wonder on 01 Nov 2019
'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 ...
Posted by Michael Wonder on 30 Oct 2019
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 ...
Posted by Michael Wonder on 24 Oct 2019
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. ...
Posted by Michael Wonder on 21 Oct 2019
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, ...
Posted by Michael Wonder on 20 Oct 2019
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 ...
Posted by Michael Wonder on 16 Oct 2019
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 ...
Posted by Michael Wonder on 15 Oct 2019
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. ...
Posted by Michael Wonder on 10 Oct 2019
'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 ...
Posted by Michael Wonder on 09 Oct 2019
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. ...
Posted by Michael Wonder on 07 Oct 2019
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 ...
Posted by Michael Wonder on 03 Oct 2019
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 ...