Blog
RSS FeedPosted by Michael Wonder on 01 Mar 2021
Nusinersen in spinal muscular atrophy: indication of considerable added benefit in children with an early onset of the disease
1 March 2021 - The treatment delays permanent ventilation of the children and extends life. In addition, the children develop more ...
Posted by Michael Wonder on 25 Feb 2021
FDA approves targeted treatment for rare Duchenne muscular dystrophy mutation
25 February 2021 - Today, the U.S. FDA granted approval for Amondys 45 (casimersen) injection for the treatment of Duchenne muscular ...
Posted by Michael Wonder on 17 Feb 2021
bluebird bio ‘baffled’ after NICE rejects beta thalassaemia gene therapy
16 February 2021 - It’s back to the drawing board for bluebird bio and its discussions with NICE, which has ...
Posted by Michael Wonder on 28 Jan 2021
Alberta announces funding for pricey gene therapy drug Zolgensma
Alberta Health Minister Tyler Shandro announced funding Wednesday for the gene therapy Zolgensma, which is used to treat toddlers with ...
Posted by Michael Wonder on 26 Jan 2021
MeiraGTx announces AAV-CNGA3 granted fast track designation by U.S. FDA for treatment of achromatopsia
26 January 2021 - MeiraGTx today announced that the U.S. FDA has granted fast track designation to its AAV-CNGA3 gene therapy ...
Posted by Michael Wonder on 19 Jan 2021
Taysha Gene Therapies receives rare paediatric disease and orphan drug designations for TSHA-105 for the treatment of epilepsy caused by SLC13A5 deficiency
19 January 2021 - Designations reinforce unmet need for treatment options for patients with rare form of genetic epilepsy. ...
Posted by Michael Wonder on 18 Jan 2021
ICER issues correction to final evidence report on new therapies for bladder cancer
15 January 2021 - After correcting an input to the cost-effectiveness model, ICER’s health-benchmark price benchmark range for nadofaragene firadenovec has ...
Posted by Michael Wonder on 21 Dec 2020
Sesen Bio submits completed biologics license application to the FDA for Vicineum and has requested priority review
21 December 2020 - BLA supported by strong Phase 3 VISTA trial data and positive analytical comparability data. ...
Posted by Michael Wonder on 21 Dec 2020
Newly approved treatment could save a young child in Edmonton with a rare disease. It costs 2.8 million dollars per dose.
19 December 2020 - Reign Johnston is a happy baby who enjoys painting as much as playing with toys. His green ...
Posted by Michael Wonder on 18 Dec 2020
Novartis receives complete response letter from U.S. FDA for inclisiran
18 December 2020 - The U.S. FDA has not raised any concerns related to the efficacy or safety of inclisiran. The ...
Posted by Michael Wonder on 18 Dec 2020
Recommendations made by the PBAC - November 2020
18 December 2020 - The recommendations from the November 2020 PBAC meeting are now available. ...
Posted by Michael Wonder on 16 Dec 2020
Health Canada approves Zolgensma, the one-time gene therapy for paediatric patients with spinal muscular atrophy
16 December 2020 - Zolgensma is designed to address the genetic root cause of SMA by replacing the missing or defective ...
Posted by Michael Wonder on 13 Dec 2020
Though promising, gene therapies face durability and reimbursement headwinds
11 December 2020 - The promise of gene therapy is to cure diseases associated with faulty or missing genes. There’s enormous ...
Posted by Michael Wonder on 04 Dec 2020
enGene receives fast track designation for EG-70 for the treatment of non-muscle invasive bladder cancer
3 December 2020 - enGene announced today that the U.S. FDA has granted fast track designation to enGene for EG-70, the ...
Posted by Michael Wonder on 19 Nov 2020
Alnylam receives approval for Oxlumo (lumasiran) in the European Union for the treatment of primary hyperoxaluria type 1 in all age groups
19 November 2020 - Oxlumo is the first therapeutic approved for the treatment of primary hyperoxaluria type 1, and the ...