Primary mitochondrial myopathy and bioenergetic disorders — indication assessment
Genetically determined impairment of oxidative phosphorylation producing myopathy, exercise intolerance or multisystem disease.
§1Assessment
§1.1Definition
Genetically determined impairment of oxidative phosphorylation producing myopathy, exercise intolerance or multisystem disease.
§1.2Anchor and additional outcomes
The Institute designates one anchor outcome per indication so that estimates for different compounds are reported on a common measure. The anchor for this indication is the first outcome below.
- Six-minute walk distance
- Primary mitochondrial myopathy symptom assessment
- Muscle phosphocreatine recovery kinetics
§1.3State of the evidence
The Institute assesses 3 compounds in this indication, drawing on 9 trial records and 3 evidence syntheses. [1,2]
References cited on this page
References are numbered in order of first citation in this document. Each superscript in the text links to its entry below.
- Szeto HH. First-in-class cardiolipin-protective compound as a therapeutic agent to restore mitochondrial bioenergetics. British Journal of Pharmacology 2014;171(8):2029–2050. doi:10.1111/bph.12461 · PMID 24117165
- D’Hondt M, Bracke N, Taevernier L, Gevaert B, Verbeke F, Wynendaele E, De Spiegeleer B. Related impurities in peptide medicines. Journal of Pharmaceutical and Biomedical Analysis 2014;101:2–30. doi:10.1016/j.jpba.2014.06.012 · PMID 25044089
Identifiers are reproduced only where the Institute holds them. Where a digital object identifier or PubMed identifier is not shown, the Institute has recorded the journal and year and has not constructed an identifier.