Blog
RSS FeedPosted 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 ...
Posted by Michael Wonder on 14 May 2020
Japan OKs public insurance coverage for SMA drug Zolgensma
13 May 2020 - A panel that advises the health minister approved on Wednesday coverage for Novartis' spinal muscular atrophy ...
Posted by Michael Wonder on 12 May 2020
Japan to set price of SMA drug Zolgensma at 167 million Yen
12 May 2020 - Japan's health ministry is planning to set the price of Novartis AG's spinal muscular atrophy drug ...
Posted by Michael Wonder on 12 May 2020
Third evaluation within the FINOSE collaboration
12 May 2020 - Together with the corresponding authorities in Finland and Norway, TLV has produced a joint evaluation report ...
Posted by Michael Wonder on 11 May 2020
Health economic assessment of gene therapy Zynteglo in transfusion-dependent beta-thalassemia
11 May 2020 - TLV has developed a health-economic knowledge base for the Zynteglo gene therapy (autologous CD34-positive cells expressing the ...
Posted by Michael Wonder on 03 May 2020
'New Zealand doesn't have help juice' - PHARMAC's decision leaves family stuck abroad
3 May 2020 - Kristie Yeoman says returning home would likely mean death for her daughter. ...
Posted by Michael Wonder on 28 Apr 2020
Spinraza could give teen a longer life, but Government says it's too expensive
28 April 2020 - A Greymouth father is battling for PHARMAC funding for a drug he hopes can help his ...
Posted by Michael Wonder on 24 Apr 2020
PHARMAC won't fund drug Spinraza to help children with spinal muscular atrophy
24 April 2020 - The parents of babies with a rare and debilitating condition will have to wait longer for ...
Posted by Michael Wonder on 28 Mar 2020
AveXis receives positive CHMP opinion for Zolgensma, the only gene therapy for spinal muscular atrophy
28 March 2020 - AveXis, a Novartis company, today announced that the CHMP of the EMA has adopted a positive ...
Posted by Michael Wonder on 19 Mar 2020
Novartis receives approval from Japanese Ministry of Health, Labour and Welfare for Zolgensma the only gene therapy for patients with spinal muscular atrophy
19 March 2020 - Reimbursement with MHLW is expected by the end of 1H20, pending agreement, Zolgensma will be available at ...
Posted by Michael Wonder on 05 Mar 2020
Gene and cell therapies continue growth in patients, financing, and costs
5 March 2020 - The number of people treated with approved gene therapies and gene-modified cell therapies like CAR-T cancer ...
Posted by Michael Wonder on 04 Mar 2020
Alnylam announces approval of Givlaari (givosiran) in the European Union for the treatment of acute hepatic porphyria in adults and adolescents
3 March 2020 - Givlaari approved based on ENVISION Phase 3 study results showing a 74% reduction in the rate of ...