Blog
RSS FeedPosted by Michael Wonder on 20 Sep 2022
AvroBio receives rare paediatric disease designation from US FDA for first gene therapy in development for cystinosis
20 September 2022 - AVR-RD-04 has previously received orphan drug designation from FDA and EMA. ...
Posted by Michael Wonder on 18 Sep 2022
bluebird bio receives FDA accelerated approval for Skysona gene therapy for early, active cerebral adrenoleukodystrophy
16 September 2022 - Skysona is the first FDA approved therapy shown to slow the progression of neurologic dysfunction in boys ...
Posted by Michael Wonder on 15 Sep 2022
Aro Biotherapeutics receives FDA rare paediatric drug designation for ABX1100 for the treatment of Pompe disease
15 September 2022 - Aro Biotherapeutics today announced that the US FDA granted rare paediatric drug designation for ABX1100, an investigational ...
Posted by Michael Wonder on 13 Sep 2022
ICER releases draft evidence report on gene therapies for haemophilia A and B
13 September 2022 - Registration now open for 20 September “Early Insights” webinar. ...
Posted by Michael Wonder on 08 Sep 2022
Silence Therapeutics announces FDA fast track designation for SLN124, a novel investigational siRNA therapy for the treatment of polycythemia vera
8 September 2022 - Designation provides potential for expedited drug development path. ...
Posted by Michael Wonder on 31 Aug 2022
CADTH ruling on Spinraza (nusinersen) extinguishes hope for adults needing treatment for spinal muscular atrophy
30 August 2022 - CADTH recommends against reimbursement and access to potentially life-altering treatment for adult spinal muscular atrophy patients, discounting ...
Posted by Michael Wonder on 30 Aug 2022
Spinal muscular atrophy community rejoices as 'life-changing' drug Spinraza approved for adults
30 August 2022 - "I have hope again," says Juanita Vernon as the 52 year old tearfully discusses her feelings about ...
Posted by Michael Wonder on 25 Aug 2022
First gene therapy for adults with severe haemophilia A, BioMarin's Roctavian (valoctocogene roxaparvovec), approved by European Commission
24 August 2022 - Maintains orphan drug designation in the EU providing 10 years of market exclusivity. ...
Posted by Michael Wonder on 23 Aug 2022
iECURE receives FDA rare paediatric disease designation for GTP-506, an investigational gene editing product candidate for the treatment of ornithine transcarbamylase deficiency
23 August 2022 - GTP-506, a potential single-dose gene editing therapy designed to restore metabolic function in patients suffering with OTC ...
Posted by Michael Wonder on 04 Aug 2022
Economic evaluation of genomic/genetic tests: a review and future directions
1 August 2022 - It has been suggested that health economists need to improve their methods in order to meet the ...
Posted by Michael Wonder on 02 Aug 2022
Korea's first gene treatment for SMA Zolgensma gets insurance benefits
2 August 2022 - Novartis Korea said Monday that it released Zolgensma, the nation's first gene replacement treatment for spinal ...
Posted by Michael Wonder on 01 Aug 2022
Listing breakthrough brings hope for people with spinal condition
1 August 2022 - Adults with debilitating spinal muscular atrophy will for the first time have access to a subsidised ...
Posted by Michael Wonder on 23 Jul 2022
Alnylam receives positive CHMP opinion for vutrisiran for the treatment of hereditary transthyretin-mediated amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy
22 July 2022 - Positive opinion based on HELIOS-A Phase 3 study. ...
Posted by Michael Wonder on 21 Jul 2022
Ultra-expensive SMA drug Zolgensma to get insurance benefits from August
21 July 2022 - Novartis' Zolgensma (onasemnogene abeparvovec), the world's most expensive drug yet to treat spinal muscular atrophy, will ...
Posted by Michael Wonder on 19 Jul 2022
ICER publishes final evidence report and policy recommendations on beti-cel gene therapy for beta thalassaemia
19 July 2022 - Independent appraisal committee unanimously determined that evidence is adequate to demonstrate that beti-cel provides a net ...