Publications

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

The Cardiolipin-Targeting Compound SBT-272 Attenuates Neurodegeneration, Delays the Onset of Neurological Signs and Extends Lifespan in Male, SOD1 G93A Transgenic Mice
By: Dennis Keefe, Guozhu Zheng, Inese Smukste, Arie Mobley, Laurent Bogdanik, Mark Bamberger

Baseline Urinary Metabolite Profiles of Primary Mitochondrial Myopathy
Subjects in the MMPOWER Study Compared with Literature Healthy Controls

By: Peter J. Oates, Jon A. Gangoiti, James Carr, Bruce A. Barshop

MMPOWER-2 Open-Label Extension Trial: 6-Month Treatment Effect on Patients with
Baseline 6MWT between 100-450 Meters

By: Richard Haas, Amy Goldstein, Jerry Vockley, Bruce H Cohen, Amel Karaa

Natural History of Disease Progression in Patients with Primary Mitochondrial Myopathy
By: Goldstein A., on behalf of the RePOWER and MMPOWER-3 Investigators

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

Elamipretide Topical Ophthalmic Solution for the Treatment of LHON: Outcomes and Efficacy
By: Rustum Karanjia, Alfredo A. Sadun

Elamipretide, a mitochondrial-targeted drug, for the treatment of vision loss in dry AMD with high risk drusen: Results of the Phase 1 ReCLAIM Study
By: Michael J. Allingham, Priyatham S. Mettu, Scott W. Cousins

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

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

MMPOWER-2 Open-Label Extension Trial: Long-term Safety and Tolerability of Elamipretide at 12 Months in Patients with Primary Mitochondrial Myopathy
By: Karaa A, Goldstein A, Haas R, Vockley J, Cohen B

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,

Placebo-Controlled Trial of Elamipretide in Primary Mitochondrial Myopathy
By: Aiudi A, DiMatteo M, Carr J

The Case for the Use of Patient and Caregiver Perception of Change Assessments in Rare Disease Clinical Trials: A Methodologic Overview
By: Marielle G. Contesse, James E. Valentine, Tracy E. Wall, Mindy G. Leffler
Published By: Springer Healthcare in Advances in Therapy March 2019

Mitochondrial Disease Patient Motivations and Barriers to Participate in Clinical Trials
By: Zarazuela Zolkipli-Cunningham, Rui Xiao, Amy Stoddart, Elizabeth M. McCormick, Amy Holberts, Natalie Burrill, Shana McCormack, Lauren Williams, Xiaoyan Wang, John L. P. Thompson, Marni J. Falk
Published By: PLoS ONE 2018

Prevalence of Nuclear and Mitochondrial DNA Mutations Related to Adult Mitochondrial Disease
By: Grainne S. Gorman, Andrew M. Schaefer, Yi Ng, Nicholas Gomez, Emma L. Blakely, Charlotte L. Alston, Catherine Feeney, Rita Horvath, Patrick Yu-Wai-Man, Patrick F. Chinnery, Robert W. Taylor, Douglass M. Turnbull, Robert McFarland

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

Cardiolipin and Mitochondrial Cristae Organization
By: Nikita Ikon and Robert O.Ryan
Elsevier- Biochimica et Biophysica Acta (BBA) – Biomembranes June 2017

Role of Cardiolipin in Mitochondrial Signaling Pathways
By: Jan Dudek
Published By: Frontiers in Cell and Developmental Biology Journal September 2017

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

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

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

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

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.

Primary Mitochondrial Myopathy Research: Patient Perspectives on Their Journey Through the Healthcare System (Abstract #: 2018 PA-0463)
By: Stanley L, Mann K, Wall T, DiMatteo M
Presented at: UMDF – Mitochondrial Medicine Conference 2018

Primary Mitochondrial Myopathy Research: Neurologists Understanding of Primary Mitochondrial Myopathies in Their Patients
By: Mann K, Stanley L, Wall T, DiMatteo M
Published By: Journal of Managed Care & Specialty Pharmacy (JMCP)

Retrospective Analysis of Healthcare Utilization in Patients with Mitochondrial Disease
By: Bruce H. Cohen, Cristy Balcells, Brian Hotchkiss, Kavita Aggarwal and Amel Karaa
Published By: Orphanet Journal of Rare Diseases

Urinary Metabolite Changes and Correlations with Six Minute Walk Test Distance in Elamipretide-treated Subjects with Mitochondrial Disease in the MMPOWER Trial Abstract #:2018 PA-0443
By: Peter J. Oates, James Carr
Presented at: UMDF – Mitochondrial Medicine 2018 Conference 2018 – Poster Presentation

Randomized dose-escalation trial of elamipretide in adults with primary mitochondrial myopathy
By: Amel Karaa, MD, Richard Haas, MD, Amy Goldstein, MD, Jerry Vockley, MD, PhD, W. Douglas Weaver, MD, and Bruce H. Cohen, MD
Published By: Wolters Kluwer Health, Inc. on behalf of the American Academy of Neurology. U.S.

Long-Term Safety and Tolerability of elamipretide: The MMPOWER-2 Open-Label Extension Trial
By: Amel Karaa, MD, Richard Haas, MD, Amy Goldstein, MD, Jerry Vockley, MD, PhD

Elamipretide Effects in Adults with Primary Mitochondrial Myopathy: Phase 2 Double-Blind, Randomized, Placebo-Controlled Crossover Trial (MMPOWER-2)
By: Amel Karaa, MD, Richard Haas, MD, Amy Goldstein, MD, Jerry Vockley, MD, PhD, and Bruce H. Cohen, MD

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