Review of Clinical Trials for Mitochondrial Disorders: 1997–2012

CoQ10 deficiencies and MNGIE: Two treatable mitochondrial disorders

(2012) Michio Hirano et al.   BIOCHIMICA ET BIOPHYSICA ACTA-GENERAL SUBJECTS

MELAS and l-arginine therapy —therapeutic timing and long term effects

(2012) Yasutoshi Koga et al.   MITOCHONDRION

Design and implementation of the first randomized controlled trial of coenzyme Q10 in children with primary mitochondrial diseases

(2012) Peter W. Stacpoole et al.   MITOCHONDRION

Restoration of impaired nitric oxide production in MELAS syndrome with citrulline and arginine supplementation

(2012) Ayman W. El-Hattab et al.   MOLECULAR GENETICS AND METABOLISM

Spectrum of neurological and survival outcomes in pyruvate dehydrogenase complex (PDC) deficiency: Lack of correlation with genotype

(2012) Suzanne D. DeBrosse et al.   MOLECULAR GENETICS AND METABOLISM

EPI-743 reverses the progression of the pediatric mitochondrial disease—Genetically defined Leigh Syndrome

(2012) Diego Martinelli et al.   MOLECULAR GENETICS AND METABOLISM

Brain uptake of Tc99m-HMPAO correlates with clinical response to the novel redox modulating agent EPI-743 in patients with mitochondrial disease

(2012) Francis G. Blankenberg et al.   MOLECULAR GENETICS AND METABOLISM

Citrulline and arginine utility in treating nitric oxide deficiency in mitochondrial disorders

(2012) Ayman W. El-Hattab et al.   MOLECULAR GENETICS AND METABOLISM

A randomized placebo-controlled trial of idebenone in Leber’s hereditary optic neuropathy

(2011) Thomas Klopstock et al.   BRAIN

Assessment of neurological efficacy of idebenone in pediatric patients with Friedreich's ataxia: data from a 6-month controlled study followed by a 12-month open-label extension study

(2011) Thomas Meier et al.   JOURNAL OF NEUROLOGY

Why are there no proven therapies for genetic mitochondrial diseases?

(2011) Peter W. Stacpoole   MITOCHONDRION

Initial experience in the treatment of inherited mitochondrial disease with EPI-743

(2011) Gregory M. Enns et al.   MOLECULAR GENETICS AND METABOLISM

MELAS and l-arginine therapy: pathophysiology of stroke-like episodes

(2010) Yasutoshi Koga et al.   Annals of the New York Academy of Sciences

A Phase 3, Double-blind, Placebo-Controlled Trial of Idebenone in Friedreich Ataxia

(2010) David R. Lynch et al.   ARCHIVES OF NEUROLOGY

The Dichloroacetate Dilemma: Environmental Hazard versus 
Therapeutic Goldmine—Both or Neither?

(2010) Peter W. Stacpoole   ENVIRONMENTAL HEALTH PERSPECTIVES

A randomized trial of coenzyme Q10in mitochondrial disorders

(2010) Elisa I. Glover et al.   MUSCLE & NERVE

Human CoQ10deficiencies

(2009) C. M. Quinzii et al.   BIOFACTORS

Idebenone: An emerging therapy for Friedreich ataxia

(2009) Thomas Meier et al.   JOURNAL OF NEUROLOGY

Treatment of mitochondrial electron transport chain disorders: A review of clinical trials over the past decade

(2009) Douglas S. Kerr   MOLECULAR GENETICS AND METABOLISM

A critical approach to the therapy of mitochondrial respiratory chain and oxidative phosphorylation diseases

(2008) Salvatore DiMauro et al.   BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE

Resistance training in patients with single, large-scale deletions of mitochondrial DNA

(2008) J. L. Murphy et al.   BRAIN

New indications and controversies in arginine therapy

(2008) David Coman et al.   CLINICAL NUTRITION

How can we treat mitochondrial encephalomyopathies? approaches to therapy

(2008) Rita Horvath et al.   Neurotherapeutics

Leigh and Leigh-Like Syndrome in Children and Adults

(2008) Josef Finsterer   PEDIATRIC NEUROLOGY