Partial pyruvate decarboxylase deficiency treated with dichloroacetate and benzoate
7 evidence topics · 2 sources
Objective
Location and study date
No evidence found.
Study design
Eligibility criteria
Enzyme deficiency
Treatment
Oral dichloroacetate dose
Study outcomes
Rationale for using serum lactate and pyruvate as the biochemical outcomes
Not applicable.
Statistical analysis description
Number of participants (Planned and analyzed)
Reported participant
Description of analysis sets
Not applicable.
Results
Participant disposition
No evidence found.
Baseline characteristics
Age at dichloroacetate administration
Efficacy results
Health-related quality of life and patient-reported outcomes
No evidence found.
Safety results
No evidence found.
Study limitations
Summary
The biochemical reductions were reported in one participant. They do not establish a comparative clinical benefit, long-term neurologic effect, or survival effect. No systematic adverse-event results are included in the retrieved evidence.