Publications

TAZPOWER Analysis: Elamipretide Significantly Improves Disease Symptomatology versus Natural History Controls in Barth Syndrome
By: Hilary J. Vernon, Ryan Manuel, John J. Jones, Jim Carr, Brittany Hornby, William Reid Thompson

Changes in Plasma and Urinary Metabolites After Elamipretide in Barth Syndrome Patients: Analyses from the TAZPOWER Study
By: David A. Brown, Hilary J. Vernon

Efficacy and Tolerability of Elamipretide in Patients with Barth Syndrome: Results from TAZPOWER, a Randomized, Double-Blind, Placebo-Controlled, Crossover and Open-Label Extension Trial
By: Hilary Vernon, MD, PhD, W. Reid Thompson, MD, Anthony Aiudi, PharmD, John J Jones, PharmD, Jim Carr, PharmD, Brittany DeCroes Hornby, PT, DPT, PCS
TAZPOWER: Biomarker Cardiolipin Ratios and Clinical Symptom Severity from a Randomized, Double-Blind, Placebo-Controlled, Crossover and Open-Label Extension Trial of Elamipretide in Barth Syndrome
By: Hilary Vernon, Brittany DeCroes Hornby, William Reid Thompson, III, Anthony Aiudi, John J. Jones, Jim Carr

TAZPOWER: Study Design of a Randomized, Double-Blind, Placebo-Controlled Crossover and Extension Trial of Elamipretide in Subjects with Barth Syndrome and Baseline Characteristics
By: Hilary Vernon, William Reid Thompson III; Anthony Aiudi; John J. Jones; Jim Carr, Meagan Elliott; Brittany DeCroes Hornby

TAZPOWER: Results of a Randomized, Double-Blind, Placebo-Controlled, Crossover and
Open-Label Extension Trial of Elamipretide in Barth Syndrome
By: Hilary Vernon, Brittany DeCroes Hornby, William Reid Thompson, III, Anthony Aiudi, John J. Jones, Jim Carr

Study Design of TAZPOWER: A Randomized, Double-Blind, Placebo-Controlled Crossover and Extension Trial of Elamipretide in Subjects with Barth Syndrome
By: Anthony Aiudi, Meaghan Elliott, Jim Carr, Brittany DeCroes Hornby, W. Reid Thompson, Hilary J. Vernon

New Targets for Monitoring and Therapy in Barth Syndrome
By: W. Reid Thompson, Brittany DeCroes, Rebecca McClellan, Jessica Rubens, Frédéric M. Vaz, Kara Kristaponis, Dimitrios Avramopoulos, Hilary J. Vernon Published By: Genetics in Medicine Journal February 2016

Bloodspot Assay Using HPLC–Tandem Mass Spectrometry for Detection of Barth Syndrome
By: Willem Kulik, Henk van Lenthe, Femke S. Stet, Riekelt H. Houtkooper, Helena Kemp, Janet E. Stone, Colin G. Steward, Ronald J. Wanders, Frédéric M. Vaz Published By: The American Association for Clinical Chemistry January 2008

Barth Syndrome: From Mitochondrial Dysfunctions Associated with Aberrant Production of Reactive Oxygen Species to Pluripotent Stem Cell Studies
By: Ana Saric, KarineAndreau1, Anne-Sophie Armand, Ian M. Møller and Patrice X. Petit Published By: Frontiers in Cell and Developmental Biology Journal January 2016

Barth Syndrome: Cellular Compensation of Mitochondrial Dysfunction and Apoptosis Inhibition Due to Changes in Cardiolipin Remodeling Linked to Tafazzin (TAZ) Gene Mutation
By: Gonzalvez F, D’Aurelio M, Boutant M, Moustapha A, Puech JP, Landes T, Arnauné-Pelloquin L, Vial G, Taleux N, Slomianny C, Wanders RJ,
Houtkooper RH, Bellenguer P, Møller IM, Gottlieb E, Vaz FM, Manfredi G, Petit PX
Published By: Elsevier- Biochimica et Biophysica Acta (BBA) – Molecular Basis of Disease August 2013

Loss of Protein Association Causes Cardiolipin Degradation in Barth Syndrome (Author Manuscript)
By: Yang Xu, Colin K.L. Phoon, Bob Berno, Kenneth D’Souza, Esthelle Hoedt, Guoan Zhang, Thomas A. Neubert, Richard M. Epand, Mindong Ren, and Michael Schlame
Published in final edited form by: Nature Chemical Biology June 2016

MTP131, a cardiolipin targeting peptide, improves mitochondrial activity in the failing human heart
By: B Stauffer, G Sparagna, S Chau, J Rodegheri-Brito, A Ambardekar, A Korst, S Miyamoto, C
Sucharov, K Chatfield, University of Colorado, Medicine/Cardiology Aurora, University of Colorado, Pediatrics/Cardiology Aurora
Published By: European Journal of Heart Failure 2016

Phase 2a Clinical Trial of Mitochondrial Protection (Elamipretide) During Stent Revascularization in Patients With Atherosclerotic
Renal Artery Stenosis
By: Ahmed Saad, MD; Sandra M.S. Herrmann, MD; Alfonso Eirin, MD; Christopher M. Ferguson, MSc; James F. Glockner, MD, PhD; Haraldur
Bjarnason, MD; Michael A. McKusick, MD; Sanjay Misra, MD; Lilach O. Lerman, MD, PhD; Stephen C. Textor, MD
Published By: American Heart Association September 2017

Elamipretide Treatment in an Infant with Sengers Syndrome

By: Rognvaldsson I., Stephensen S.S., Oskarsson G., Gunnarsdottir B.B., Marelsson S., Jonsson J.J., Bjornsson H.T., Department of Pediatrics,
Barnaspitali Hringsins, Reykjvaik, Iceland; Department of Genetics and Molecular Medicine, Landspitali University Hospital, Reykjavik, Iceland; Department of Biochemistry and
Molecular Biology, Faculty of Medicine, University of Iceland; McKusick-Nathans, Institute of Genetics Medicine, Johns Hopkins University, Baltimore, MD USA

Elamipretide in Patients with Barth Syndrome: A Randomized, Double Blind, Placebo-Controlled Clinical Trial Followed by 36-Week Open-Label Extension

By: William Reid Thompson, Ryan Manuel, Anthony Aiudi, John J. Jones, Jim Carr, Brittany Hornby, Hilary J. Vernon

Role of Cardiolipin Alterations in Mitochondrial Dysfunction and Disease
By: Adam J. Chicco, and Genevieve C. Sparagna
Published By: The American Physiological Society January 2007

Serendipity and the Discovery of Novel Compounds That Restore Mitochondrial Plasticity
By: HH Szeto and AV Birk
Published By: American Society for Clinical Pharmacology & Therapeutics December 2014

Guiding Ageing Research Into Maturity
By: Dr. David Marcinek
Available By: Scientia -Celebrating Cell Biology and Biochemistry

Mitochondrial-targeted peptide rapidly improves mitochondrial energetics and skeletal muscle performance in aged mice
By: Michael P. Siegel, Shane E. Kruse, Justin M. Percival, Jorming Goh, Collin C. White, Heather C. Hopkins, Terrance J. Kavanagh, Hazel H.
Szeto, Peter S. Rabinovitch and David J. Marcinek
Published By: Anatomical Society Journal – The Aging Cell.

Elamipretide Improves Skeletal Muscle Function in Elderly Subjects: Results from a Randomized, Double-Blind, Placebo-Controlled, Single IV-Dose Study
By: Conley KE, PhD, Liu Z, PhD, Ali AS, Amory JK, MD, Robertson HL, MD, Goss C, Shankland EG, PhD, Marcinek DM, PhD, and Roshanravan B, MD
Presented at: Heart Failure Society of America 2016.

Effects of the Mitochondria-Targeted Drug Elamipretide on Leakage-Independent Vision Loss in Fellow Eyes with Neovascular AMD in the ReCLAIM Study
By: Priyatham S. Mettu, Michael J. Allingham, Scott W. Cousins

A mitochondrial therapeutic reverses visual decline in mouse models of diabetes
By: Nazia M. Alam, William C. Mills IV, Aimee A. Wong, Robert M. Douglas, Hazel H. Szeto and Glen T. Prusky
Published by: The Company of Biologists Ltd | Disease Models & Mechanisms (2015)

Role of Mitochondrial Dysfunction in Dry Age-Related Macular Degeneration
By: Scott W. Cousins, MD
Published By: Retina Today May/June 2015