Blog
RSS FeedPosted by Michael Wonder on 18 Sep 2019
'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 ...
Posted by Michael Wonder on 17 Sep 2019
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 ...
Posted by Michael Wonder on 13 Sep 2019
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 ...
Posted by Michael Wonder on 12 Sep 2019
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 ...
Posted by Michael Wonder on 12 Sep 2019
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 ...
Posted by Michael Wonder on 11 Sep 2019
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 ...
Posted by Michael Wonder on 08 Sep 2019
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 ...
Posted by Michael Wonder on 04 Sep 2019
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 ...
Posted by Michael Wonder on 30 Aug 2019
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) ...
Posted by Michael Wonder on 28 Aug 2019
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 ...
Posted by Michael Wonder on 28 Aug 2019
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 ...
Posted by Michael Wonder on 27 Aug 2019
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 ...
Posted by Michael Wonder on 23 Aug 2019
‘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 ...
Posted by Michael Wonder on 22 Aug 2019
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 ...
Posted by Michael Wonder on 20 Aug 2019
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 ...