Clinical Trial Update: Use of Ravicti™ in Patients With MCAD Deficiency With the 985A>G (K304E) Mutation [Phase 1]

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Summary

Phase 1 trial update evaluating Ravicti in patients with MCAD deficiency carrying the 985A>G (K304E) mutation has completed with results posted.

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Diseases / indications

• Medium-chain Acyl-coa Dehydrogenase Deficiency

Drugs mentioned

• Ravicti (glycerol phenylbutyrate)

Snippet

Sponsor: University of Pittsburgh | Conditions: Medium-chain Acyl-CoA Dehydrogenase (MCAD) Deficiency | Phase 1 | Status: COMPLETED | Results posted: 2017-07-11