Publications

Barth Syndrome

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

Mechanism of Action

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

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.

Neurology

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

Ophthalmic/Dry Age-Related Macular Degeneration/Leber Hereditary Optic Neuropathy

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

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

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