Gene Therapy for Sickle Cell Slows
This is a Vertex news story, published by Yahoo, that relates primarily to Oxbryta news.
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National Comprehensive Sickle Cell Disease ProgramReuters
•Why gene therapy for sickle cell is slow to catch on with patients
80% Informative
New gene therapies may provide long-term relief to some of the 100,000 Americans who suffer from sickle cell disease.
Take-up for potentially life-changing treatments is proving even slower than expected, experts say.
Younger patients reluctant to add more medical burden to their lives.
8 million people are estimated to have sicklecell disease, an inherited disorder.
Until now, the only potential cure for sickle cell disease was a bone marrow transplant.
Pfizer said it was withdrawing its sickle-cell disease treatment, Oxbryta , citing risks of a painful complication and deaths.
Vertex partnered with CRISPR Therapeuticsto develop its $2.2 million therapy Casgevy , the first U.S.-approved treatment.
Both Vertex and Bluebird have programs to help with payment for fertility services.
Fertility services are not available to patients in the federal government's Medicaid plan for low-income individuals.
Medicaid has proposed a pilot program starting next year that would include some fertility services, but Vertex is challenging the government's policy in court.
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