Why are there no proven therapies for genetic mitochondrial diseases?

被引：19

作者：

Stacpoole, Peter W. ^{[1
,2
]}

机构：

[1] Univ FL, Dept Med, Coll Med, Div Endocrinol & Metab, Gainesville, FL USA

[2] Univ FL, Dept Biochem & Mol Biol, Coll Med, Gainesville, FL USA

来源：

MITOCHONDRION | 2011年 / 11卷 / 05期

关键词：

Clinical trials; Coenzyme Q(10); Dichloroacetate; Mitochondria; Mitochondrial diseases; CONGENITAL LACTIC-ACIDOSIS; CONTROLLED CLINICAL-TRIAL; PLACEBO-CONTROLLED TRIAL; COENZYME Q(10); HUNTINGTONS-DISEASE; CEREBELLAR-ATAXIA; ENERGY-METABOLISM; DICHLOROACETATE; DEFICIENCY; CREATINE;

D O I：

10.1016/j.mito.2011.05.002

中图分类号：

Q2 [细胞生物学];

学科分类号：

071009 ; 090102 ;

摘要：

Although mitochondrial disease research in general is robust, adequate treatment of these life-threatening conditions has lagged, partly because of a persistence of clinical anecdotes as substitutes for scientifically and ethically rigorous clinical trials. Here I summarize the key lessons learned from some of the "first generation" of randomized controlled trials for genetic mitochondrial diseases and suggest how future trials may benefit from both past experience and exciting new resources available for patient-oriented research and training in this field. (C) 2011 Elsevier B.V. and Mitochondria Research Society. All rights reserved.

引用

页码：679 / 685

页数：7

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