Both AAV vectors and genome editing novel technologies are being brought to bear in this unique and experimental gene therapy.
The applications are for the company's investigational gene therapy candidate for adults with hemophilia A.
The data presented at ISTH reflected marked improvements in FVIII half-life amongst study participants.
Bayer has announced a recall of two lots of Kogenate® FS 2000 IU vials.
uniQure's gene therapy candidate AMT-061 is being developed for individuals with severe and moderately severe hemophilia B.
The final data was presented for the first time at a symposium at the XXVII Congress of the International Society on Thrombosis and Haemostasis (ISTH).
Much of the data was gleaned from the HAVEN clinical trial program.
The findings presented at ISTH were based on an ongoing phase 1/2 clinical trial in patients with severe hemophilia A.
NHF's Connections for Learning program brings families education and support.
Protections for pre-existing conditions and coverage standards at risk
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