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A Single Patient Treatment Use of UX007 for Adult Malonyl-coA Decarboxylase Deficiency

Overview

About this study

This plan is intended for the use of UX007 (triheptanoin) in the treatment of a single patient with adult Malonyl-coA decarboxylase deficiency.

 

 

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential participation.

Inclusion Criteria:

  • Treatment of a single patient with adult Malonyl-coA decarboxylase deficiency

Participating Mayo Clinic locations

Study statuses change often. Please contact the study team for the most up-to-date information regarding possible participation.

Mayo Clinic Location Status Contact

Rochester, Minn.

Mayo Clinic principal investigator

David Burkholder, M.D.

Closed for enrollment

Contact information:

Cheolsu Shin M.D.

(507) 284-9653

shin@mayo.edu

More information

Publications

Publications are currently not available

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CLS-20307294

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