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Gene Therapy Resources

Gene therapies represent an exciting new way to treat disease. Naturally, stakeholders have a lot of questions about these transformative therapies – from how they work and how they’re made to what diseases they’ll treat and whether they’ll be covered by insurance. 

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Here we have gathered some of the most informative resources on gene therapies from our advisory council members and beyond. We hope this one-stop-shop is a helpful guide to learning about gene therapies.

What are Gene Therapies? 

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Background on the science behind gene therapies and how they’re made.

The American Society of Gene and Cell Therapy

Sarepta Therapeutics

The American Society of Gene and Cell Therapy

What Is Gene Therapy? How Does It Work?

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U.S. Food & Drug Administration

The American Society of Gene and Cell Therapy

genehome

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bluebird bio

The Value of Gene Therapies

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Value for Whom? Incorporating Patient Perspectives into Value
Assessment for Novel Cell and Gene Therapies

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EveryLife Foundation Position Paper developed by Health Advances in collaboration with the Partnership to Improve Patient Care

Solutions to Access Barriers

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Improving Access to
Newborn Screening

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Institute for Gene Therapies

Ensuring Patient Access to Gene Therapies

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Institute for Gene Therapies

IGT Comments on Policy Proposals

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policy-briefs
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