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<span>New Path For a Gene Therapy Trial At NIH For a Rare Metabolic Disease</span>
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<h1 class="page-title">New Path for a Gene Therapy Trial at NIH for a Rare Metabolic Disease</h1>
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<p>Scientists at NCATS and other NIH institutes and centers are teaming up to advance a gene therapy treatment for MMA, a rare and potentially life-threatening genetic disorder.</p>
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Program Areas
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Bespoke Gene Therapy Consortium (BGTC)
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Our Impact on Clinical Trials
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Our Impact on Drug Discovery and Development
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Our Impact on Rare Diseases
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Platform Vector Gene Therapy (PaVe-GT)
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