New Drug Application on Track for LCFAOD A Rare Autosomal Recessive Disease

Post author:Rose Duesterwald
Post published:April 2, 2019
Post category:Benign congenital hypotonia Cardiomyopathy- X-linked- fatal infantile Triglyceride storage disease with impaired long-chain fatty acid oxidation

A recent article in the publication CheckOrphan announced encouraging results of a long term extension study sponsored by Ultragenyx involving UX007 (triheptanoin) in patients with long-chain fatty acid oxidation disorders…

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New Drug Application on Track for LCFAOD A Rare Autosomal Recessive Disease

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