Product information and disease description Place of product in therapy Approaches to treatment Current treatment options and standard of care

Limitations of current therapies

5 evidence topics · 6 sources

Summary

Ketogenic dietary treatment must be individualized because tolerance varies. A clinical-practice review identified 40 studies describing 129 participants, predominantly case reports or case series. A separate treatment scoping review included seven preclinical and ten human studies and concluded that most interventions had not been rigorously investigated. These evidence designs limit the certainty of comparative treatment effects.

Unmet treatment need

“There are currently no FDA-approved therapies for PDCD.” (opens the source at this quote in a new tab)

Cochrane review: mitochondrial disorders, literature search through July 2011

“three trials in children showed a statistically significant improvement in biochemical measurements but no clinical benefit on physiological measurements and exercise tests” (opens the source at this quote in a new tab)

PDCD scoping review and case series

“We reviewed and summarized findings from seven preclinical studies and ten human studies” (opens the source at this quote in a new tab)
“However, most interventions have yet to be rigorously investigated.” (opens the source at this quote in a new tab)

Natural-history cohort: absence of demonstrated benefit for any intervention

“Five subjects had used DCA, and one family reported that it was “very helpful”.” (opens the source at this quote in a new tab)
“Subjective retrospective assessments of the benefits of these various interventions were most favorable for ketogenic diet treatment, but we cannot confidently determine from these data benefit or lack thereof for any of these interventions.” (opens the source at this quote in a new tab)
“This retrospective correlation of PDC activity, mutations, and outcomes was not designed to assess whether specific treatments were effective in improving outcomes.” (opens the source at this quote in a new tab)