Uptake of new hemophilia gene therapies slow as field assesses options: High cost, logistical issues and the prospect of potential treatment advances are holding ...
reuters.com/business/healthcare-pharmaceuticals/uptake-new-hemophilia-gene-therapies-slow-field-assesses-options-2023-12-15/
$2.9 million gene therapy for severe hemophilia is approved by FDA: U.S. health regulators have approved a gene therapy for the most common form of hemophilia
abcnews.go.com/Business/wireStory/gene-therapy-severe-hemophilia-approved-fda-100508774
Quick Glance: BioMarin receives FDA approval for gene therapy in hemophilia A
- BioMarin announces FDA approval of gene therapy for severe hemophilia A.
- Roctavian gene therapy offers an alternative to regular injections of missing blood proteins.
- Roctavian is priced at $2.9 million.
- Pivotal trial results show that Roctavian reduces bleeding events, but its long-term durability is unknown.
Gene therapy can transform life for people with hemophilia. But some patients don’t want it.
bostonglobe.com/2023/01/29/business/gene-therapy-can-transform-life-people-with-hemophilia-some-patients-dont-want-it/
BioMarin’s Gene Therapy Cuts Annual Bleeds by 80% Over 3 Years: BioMarin Pharmaceutical Inc.’s gene therapy Roctavian reduced the annualized rate of bleeding in ...
bloomberg.com/news/articles/2023-01-08/biomarin-s-gene-therapy-cuts-annual-bleeds-by-80-over-3-years
First gene therapy for hemophilia A before approval: Amsterdam - The European Medicines Agency (EMA) has given the go-ahead for the first gene therapy for ...
aerzteblatt.de/nachrichten/135470/Erste-Gentherapie-der-Haemophilie-A-vor-der-Zulassung
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