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  2. Dystrophin Education For DMD - About Gene Therapy for DMD

    Increasing Dystrophin May Slow The Progression Of Duchenne. Learn More About DMD Treatment. Learn About The Different Ways To Increase Dystrophin In People With Duchenne

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  3. Living With Fabry Disease? - See If You Qualify Today

    Investigational Gene Therapy to Treat Fabry Disease. Complete The Screener Questionnaire and See If You Qualify Now!

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    • TIME100 Health: Jerry Mendell

      TIME100 Health: Jerry Mendell

      Time via Yahoo News· 2 hours ago

      Symptoms typically begin when children are 3 to 6 years old, and get progressively worse, ultimately leading to breathing problems and heart issues as...

    • Daniel Skovronsky

      Daniel Skovronsky

      Time Magazine· 2 hours ago

      Daniel Skovronsky, its chief scientific officer. Skovronsky’s efforts are pushing the company to the head of the pack when it comes to drugs addressing...

    • Bobby Gaspar

      Bobby Gaspar

      Time Magazine· 2 hours ago

      Dr. Bobby Gaspar, CEO of Orchard Therapeutics, has the dubious honor of making the most expensive medicine—at $4.5 million per dose—currently on the...

    • Peter Marks

      Peter Marks

      Time Magazine· 2 hours ago

      In 2023, the U.S. Food and Drug Administration (FDA) brought a number of never-before-seen treatments to market, approving the first gene therapies using...

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    related to: gene therapy

  2. Dystrophin Education For DMD - About Gene Therapy for DMD

    Increasing Dystrophin May Slow The Progression Of Duchenne. Learn More About DMD Treatment. Learn About The Different Ways To Increase Dystrophin In People With Duchenne

    Types: DMD Education, Duchenne-Opedia, Patient Stories, Helpful Materials

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