Blog
RSS FeedPosted 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 ...
Posted by Michael Wonder on 05 Jul 2020
Clock ticking on Edmonton family's hope for $2.8 million drug treatment for toddler son
3 July 2020 - Family hoping Alberta Health will pay for new drug that must be administered by mid-July. ...
Posted by Michael Wonder on 01 Jul 2020
Life-changing drug remains unfunded
1 July 2020 - Knowing his body is slowly deteriorating is ‘‘the scariest feeling ever’’. ...
Posted by Michael Wonder on 24 Jun 2020
Germany first in EU to get Novartis’ SMA gene therapy, costing almost 2 million euros
24 June 2020 - Novartis is set to launch its one-off gene therapy Zolgensma for the ultra-rare muscle-wasting disease spinal ...
Posted by Michael Wonder on 15 Jun 2020
Teen with spinal muscular atrophy campaigns for wonder drug to survive
15 June 2020 - Taylor Fincham is one of 35 Kiwi children who suffer from spinal muscular atrophy. ...
Posted by Michael Wonder on 11 Jun 2020
Cell and gene therapies: FDA official on COVID-19 impact
10 June 2020 - Speaking at a session on cell and gene therapies at BIO Digital on Monday, a top US ...
Posted by Michael Wonder on 10 Jun 2020
Hawke's Bay mum's heartbreak as PHARMAC refuses spinal muscular atrophy drug for her two kids
9 June 2020 - Skylah-Rose Shaw 8 and her brother Zayden Shaw 10 suffer from spinal muscular atrophy a disease that ...
Posted by Michael Wonder on 27 May 2020
Alnylam announces U.S. FDA has granted priority review of the lumasiran new drug application for the treatment of primary hyperoxaluria type 1
26 May 2020 - PDUFA date set for 3 December 2020. ...
Posted by Michael Wonder on 22 May 2020
Passage Bio receives rare paediatric disease designation for PBGM01 for patients with GM1 gangliosidosis
21 May 2020 - PBGM01 to enter the clinic in fourth quarter of 2020. ...
Posted by Michael Wonder on 20 May 2020
B.C. teen with rare disease wins fight to receive costly drug from province
19 May 2020 - Health ministry won't explain change of heart in granting Miles Ambridge treatment for spinal muscular atrophy. ...
Posted by Michael Wonder on 19 May 2020
AveXis receives EC approval and activates “Day One” access program for Zolgensma, the only gene therapy for spinal muscular atrophy
19 May 2020 - Zolgensma (onasemnogene abeparvovec) is conditionally approved in Europe for the treatment of patients with spinal muscular atrophy ...