Blog
RSS FeedPosted by Michael Wonder on 27 May 2021
Ireland an outlier without access to new cell and gene therapy, report says
27 May 2021 - Upfront costs high but treatments may cut long-term expense and boost patient results. ...
Posted by Michael Wonder on 26 May 2021
Colchester baby's parents feel 'abandoned' over £1.7 million drug
25 May 2021 - A baby with a fatal genetic condition has been "abandoned" in a "race against time" for ...
Posted by Michael Wonder on 25 May 2021
European Medicines Agency grants BioMarin's request for accelerated assessment of valoctocogene roxaparvovec for treatment of severe haemophilia A
24 May 2021 - MAA resubmission on track for June 2021. ...
Posted by Michael Wonder on 23 May 2021
First gene therapy to treat children with rare inherited neurological disease
21 May 2021 - The EMA has recommended granting a marketing authorisation in the European Union for the gene therapy ...
Posted by Michael Wonder on 19 May 2021
FDA: flexibility okay for gaps in gene therapy trials
18 May 2021 - The US FDA is not amenable to changing study outcome measures or sample sizes for gene therapy ...
Posted by Michael Wonder on 14 May 2021
BridgeBio Pharma receives FDA fast track designation for investigational gene therapy for congenital adrenal hyperplasia
14 May 2021 - BridgeBio Pharma today announced that the U.S. FDA granted fast track designation to BBP-631, an investigational adeno-associated ...
Posted by Michael Wonder on 10 May 2021
Dutch advisory group says Zolgensma shouldn’t be covered unless Novartis halves price
10 May 2021 - In a challenge to Novartis, a Dutch advisory body has recommended that the government not cover ...
Posted by Michael Wonder on 07 May 2021
World’s most expensive drug may not be covered by Dutch insurance
7 May 2021 - A drug to treat a rare muscle disease which affects some 20 children in the Netherlands ...
Posted by Michael Wonder on 05 May 2021
PHARMAC asked to fund drug for children with spinal muscular atrophy after it changes four year old's life
5 May 2021 - Four year old Charlotte Bond couldn’t roll over by herself two years ago. ...
Posted by Michael Wonder on 04 May 2021
NICE announces more people eligible for nusinersen sodium following review of Managed Access Agreement
4 May 2021 - NICE has today announced that more people with the rare genetic disorder spinal muscular atrophy are to ...
Posted by Michael Wonder on 22 Apr 2021
TGA publishes AusPAR for Zolgensma
22 April 2021 - Rapid publication following approval. ...
Posted by Michael Wonder on 21 Apr 2021
Additional analysis of Luxturna in the treatment of visual impairment caused by hereditary eye disease
21 April 2021 - TLV has made a supplementary analysis to the previous evaluation of Luxturna, as Novartis has submitted new ...
Posted by Michael Wonder on 20 Apr 2021
LEXEO Therapeutics announces FDA fast track designation granted to LX1001 for the treatment of APOE4 associated Alzheimer’s disease
20 April 2021 - LX1001 is the first investigational gene therapy being evaluated to address the underlying genetics of Alzheimer’s disease. ...
Posted by Michael Wonder on 29 Mar 2021
Alberta family waiting on approval for expensive, life-saving drug for young boy
23 March 2021 - Reign Johnston turned 2 on March 5. One of his grandpas and an uncle came to ...
Posted by Michael Wonder on 27 Mar 2021
FDA approves first cell-based gene therapy for adult patients with multiple myeloma
27 March 2021 - The U.S. FDA approved Abecma (idecabtagene vicleucel), a cell-based gene therapy to treat adult patients with multiple ...