July 12, 2021 10:20 AM EDTUpdated 11:20 AM

Switching focus in the brain, researchers herald a possible gene therapy breakthrough in treating an ultra-rare disease

John Carroll

Editor & Founder

A group of investigators testing a gene therapy for ultra-rare cases of aromatic amino acid decarboxylase deficiency has seen some remarkable results in their first …

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Switching focus in the brain, researchers herald a possible gene therapy breakthrough in treating an ultra-rare disease

John Carroll

Editor & Founder

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Switch­ing fo­cus in the brain, re­searchers her­ald a pos­si­ble gene ther­a­py break­through in treat­ing an ul­tra-rare dis­ease

John Carroll

Editor & Founder

Sign up to read this article for free.

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Switching focus in the brain, researchers herald a possible gene therapy breakthrough in treating an ultra-rare disease

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Applied Therapeutics' rare disease drug gets a CRL

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FDA is looking into hematology risks after patients take Bluebird's Skysona gene therapy

Outlook's shares crash as wet AMD drug flunks Phase 3 test

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