Blog
RSS FeedPosted by Michael Wonder on 21 Jan 2020
Genprex receives U.S. FDA fast track designation for gene therapy that targets lung cancer
21 January 2020 - Genprex today announced that the U.S FDA has granted fast track Designation for Genprex’s Oncoprex immunogene ...
Posted by Michael Wonder on 17 Jan 2020
BioMarin explores pricing experimental gene therapy at $2 million to $3 million
16 January 2020 - Valrox would be first gene therapy approved in U.S. for inherited type of haemophilia. ...
Posted by Michael Wonder on 14 Jan 2020
B.C. funding decision on pricey drug leaves teen with rare disease facing 'scary' future
13 January 2020 - Miles Ambridge just misses cut-off age to access Spinraza, a potentially life-changing treatment. ...
Posted by Michael Wonder on 14 Jan 2020
Roche aims to 'underwhelm' on SMA drug price to challenge rivals
14 January 2020 - Swiss drug maker Roche plans to price its oral spinal muscular atrophy drug risdiplam aggressively to challenge ...
Posted by Michael Wonder on 14 Jan 2020
PTC Therapeutics provides corporate update
13 January 2020 - PTC-AADC MAA submitted; BLA now expected to be submitted in 2Q 2020. ...
Posted by Michael Wonder on 12 Jan 2020
At 16, she’s a pioneer in the fight to cure sickle cell disease
11 January 2020 - Helen Obando is the youngest person ever to get a gene therapy that scientists hope will cure ...
Posted by Michael Wonder on 09 Jan 2020
New CADTH process for cell and gene therapies
9 January 2020 - CADTH has undertaken an internal review of our processes for drugs and devices, and established a revised ...
Posted by Michael Wonder on 09 Jan 2020
Aruvant receives FDA rare paediatric disease designation for ARU-1801 for the treatment of sickle cell disease
8 January 2020 - ARU-1801 is being developed as a potential one-time treatment for patients suffering from sickle cell disease and ...
Posted by Michael Wonder on 06 Jan 2020
ICER posts draft scoping document for the assessment of treatments for beta thalassaemia
6 January 2020 - Document open to public comment until 27 January 2020. ...
Posted by Michael Wonder on 06 Jan 2020
Canadian parents of babies with rare deadly disease look to Novartis treatment lottery
5 January 2020 - Canadian babies with a rare and often fatal muscle-wasting disease may soon have access to a ...
Posted by Michael Wonder on 04 Jan 2020
ICER to assess gene therapy for haemophilia A
3 January 2020 - Report will be subject of New England CEPAC meeting in August 2020; open Input now being accepted ...
Posted by Michael Wonder on 04 Jan 2020
NICE refuses Akcea’s Waylivra
3 January 2020 - NICE has issued an Evaluation Consultation Document, refusing Waylivra (volanesorsen) as a treatment for familial chylomicronaemia ...
Posted by Michael Wonder on 02 Jan 2020
Toronto parents of baby needing $2.8 million drug turn to Swiss drug maker's dose lottery as a last resort
31 December 2019 - Critics call on Ottawa to improve access to expensive drugs that treat rare diseases. ...
Posted by Michael Wonder on 24 Dec 2019
BioMarin submits biologics license application to U.S. FDA for valoctocogene roxaparvovec to treat haemophilia A
23 December 2019 - First marketing application submission in U.S. for gene therapy directed at any type of haemophilia. ...
Posted by Michael Wonder on 24 Dec 2019
European Medicines Agency validates BioMarin's marketing authorisation application for valoctocogene roxaparvovec to treat severe haemophilia A
23 December 2019 - Potential first gene therapy in Europe directed at any type of haemophilia. ...