Blog
RSS FeedPosted by Michael Wonder on 14 Sep 2020
Stranded mum with dying daughter makes desperate plea to Jacinda Ardern
13 September 2020 - A distraught mum stuck in New Zealand with her dying daughter Stella amid the COVID pandemic ...
Posted by Michael Wonder on 12 Sep 2020
Kiwi mum pleads with PHARMAC to fund life-changing drug Spinraza for her two children
11 September 2020 - The mother of two young boys with a rare disease is begging PHARMAC to fund a ...
Posted by Michael Wonder on 12 Sep 2020
NICE widens its Zolgensma appraisal due to European Marketing Authorisation
11 September 2020 - England’s NICE is expanding its appraisal of Zolgensma, a gene therapy for spinal muscular atrophy, according ...
Posted by Michael Wonder on 10 Sep 2020
EU review extended for BioMarin's gene therapy Valrox
10 September 2020 - European regulators have requested more data to review the marketing application for BioMarin's haemophilia A gene ...
Posted by Michael Wonder on 28 Aug 2020
Employers are planning how to blunt the cost of gene therapies, pricey new specialty drugs
27 August 2020 - As the pharmaceutical industry develops increasingly expensive medicines – notably, those costing seven figures – a ...
Posted by Michael Wonder on 28 Aug 2020
Taysha Gene Therapies receives orphan drug designation and rare paediatric disease designation for TSHA-101 for GM2 gangliosidosis
27 August 2020 - TSHA-101 anticipated to advance into the clinic by the end of 2020. ...
Posted by Michael Wonder on 19 Aug 2020
BioMarin receives complete response letter from FDA for valoctocogene roxaparvovec gene therapy for severe haemophilia A
19 August 2020 - FDA introduces new recommendation for 2 year annualised bleeding rate as primary outcome for on-going Phase 3 ...
Posted by Michael Wonder on 15 Aug 2020
Family battles PHARMAC and eyes Australian move to get 'miracle drug'
15 August 2020 - When Lani McLeod found out her toddler would never walk, she was gutted. ...
Posted by Michael Wonder on 13 Aug 2020
4D Molecular Therapeutics receives FDA fast track designation for 4D-310 gene therapy for treatment of Fabry disease
13 August 2020 - 4D Molecular Therapeutics announced that the U.S. FDA has granted fast track designation to 4D-310 for ...
Posted by Michael Wonder on 13 Aug 2020
TGA approves new gene therapy from Novartis
13 August 2020 - Luxturna has been for the treatment of patients with inherited retinal dystrophy caused by pathological biallelic RPE65 ...
Posted by Michael Wonder on 10 Aug 2020
B.C. family nearing finish in race to raise $2.8 million to treat baby's rare disease
8 August 2020 - Parents hope funds will pay for expensive therapy not approved in Canada for type 1 spinal muscular ...
Posted by Michael Wonder on 26 Jul 2020
'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 ...
Posted by Michael Wonder on 24 Jul 2020
Sarepta Therapeutics receives fast track designation for SRP-9001 micro-dystrophin gene therapy for the treatment of Duchenne muscular dystrophy
24 July 2020 - Sarepta Therapeutics today announced that the U.S. FDA has granted fast track designation to SRP-9001 (AAVrh74.MHCK7.micro-dystrophin). ...
Posted by Michael Wonder on 20 Jul 2020
Gene therapy shows promise for haemophilia, but could be most expensive U.S. drug ever
20 June 2020 - Jack Grehan, who was born with hemophilia, used to inject himself every couple of days with ...
Posted by Michael Wonder on 18 Jul 2020
'Heartbreaking': Alberta families describe waiting for life-changing drug to be made available in Canada
17 July 2020 - More families are hoping a one-time treatment option for a rare condition will be made available ...