Tag: gene therapy

Stories

Advocacy Groups Funding DMD Gene Therapy Trial

Gene therapy for rare diseases continues to make headlines. The latest clinical trial involving gene therapy is one for Duchenne muscular dystrophy (DMD). That trial recently dosed its first patient at the Nationwide Children’s Hospital in Columbus, Ohio.

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Gene Therapy and Hemophilia A

The results of a phase 1/2, clinical study looking at the safety and efficacy of gene therapy to treat 9 adults with severe hemophilia A was recently published in the New England Journal of Medicine (NEJM). And the results are very promising.

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FDA Approves Gene Therapy for Rare Eye Condition

Gene therapy is now a reality. At least in the United States. Today, the U.S. Food and Drug Administration (FDA) approved the gene therapy Luxturna (voretigene neparvovec-rzyl) to treat patients with a rare form of blindness – biallelic RPE65 mutation-associated retinal dystrophy.

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Gene Therapy and Hemophilia B

This week in the  New England Journal of Medicine, Lindsey George, MD and colleagues report on their Phase 1/2 study assessing the safety and efficacy of gene therapy to treat patients with hemophilia B. And the results are very impressive.

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