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Irish baby among world’s first to receive experimental gene therapy

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The Next Frontier in Newborn Screening and Why It Matters for Canavan Families

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The Rare Disease Conversation Is Getting Louder. Here’s Where Myrtelle Stands.

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Myrtelle Announces Nature Medicine Publication of Interim Results from Its Phase 1/2 Clinical Trial of Investigational Gene Therapy rAAV-Olig001-ASPA for Canavan Disease

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