gene therapy

Groundbreaking gene therapies in store for hemophiliaTreatment failures can result in a spectrum of problems for patients with hemophilia. Here’s some guidance on choosing the right therapy.
EB’s great hopePediatricians respond to the first successful gene therapy treatment for epidermolysis bullosa.
New hemophilia B gene therapy appears effectiveA recent trial showed that a single intravenous infusion of a vector expressing a high specific-activity factor IX gene safely produces a sustained, effective level of factor IX coagulant activity in patients with hemophilia B.
Blindness gene therapy price tag stirs controversyA patient advocacy group is criticizing the hefty price tag of a new treatment for blindness.
Gene therapy benefits for retinal dystrophies persist through trialResults from 3 years of follow-up in the phase III trial investigating treatment with adeno-associated viral vector delivery of human RPE65 (voretigene neparvovec, Spark Therapeutics) show this gene therapy has an acceptable safety profile.
Tisagenlecleucel (Kymriah) for Acute Lymphoblastic Leukemia
Tisagenlecleucel (Kymriah) for Acute Lymphoblastic LeukemiaA new era of treatment has arrived—here’s what you need to know.
Game-changing gene therapies present cost problems for health execsCAR-T therapies have high price tags so it’s important to understand your patient population and budget impact.
Four ways health execs can prep for costly CAR-T cell therapiesWith the high efficacy and dire diagnosis, there will be demand for both Kymriah and Yescarta. Here’s how to manage the monetary impact.
First gene therapy for cancer approved: What health execs should knowThe first therapy based on gene transfer has been approved by the FDA. What’s next for healthcare executives?
Gene therapy trial for blistering skin condition advancing to next stage
Gene therapy trial for blistering skin condition advancing to next stageFCX-007 could be the first major medical advance for recessive dystrophic epidermolysis bullosa, or RDEB, in decades.