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Frequently Asked Questions
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Frequently Asked Questions

Find answers to the most common questions around technology commercialization.

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Muscular Dystrophy Research

Dr. Jerry Mendell and his team have discovered that gene therapy can treat the underlying cause of muscular dystrophy - something that other drugs and medications can't currently do.

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March 2012

Dr. Thomas Voit discusses gamma sarcoglycan gene therapy.

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January 2013

Dr. James Ervasti discusses stability of therapeutic forms of dystrophin.

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September 2010

Dr. Federica Montanaro discusses cardiomyopathy in becker muscular dystrophy.

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February 2012

Dr. Burghes discusses antisense oligomer treatment in an SMA mouse model.

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May 2012

Dr. Hugh Allen discusses cardiomyopathy in duchenne muscular dystrophy.

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September 2011

Dr. Jill Rafael-Fortney discusses the effects of Lisinopril and Spironolactone on DMD mice.

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December 2012

Dr. Federica Montanaro discusses new cardiac dystrophin associated proteins.

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January 2015

Dr. Louise Rodino-Klapac discusses dysferlin overlap vectors to restore function in dysferlinopathies.

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