Publications by authors named "Sharon Hesterlee"

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Consensus recommendations and considerations for the delivery and monitoring of gene therapy in patients with Duchenne muscular dystrophy.
Jodi M Wolff, Nora Capocci, Evrim Atas, Diana X Bharucha-Goebel, John F Brandsema, Sharon Hesterlee

Neuromuscul Disord· September 2025

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Meeting Report: 2023 Muscular Dystrophy Association Summit on 'Safety and Challenges in Gene Therapy of Neuromuscular Diseases'.
Angela Lek, Evrim Atas, Brian Lin, Sharon E Hesterlee, Jordan K Abbott

J Neuromuscul Dis· September 2024

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Meeting Report: 2022 Muscular Dystrophy Association Summit on 'Safety and Challenges in Gene Transfer Therapy'.
Angela Lek, Evrim Atas, Sharon E Hesterlee, Barry J Byrne, Carsten G Bönnemann

J Neuromuscul Dis· May 2023

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The Muscular Dystrophy Association's neuroMuscular ObserVational Research Data Hub (MOVR): Design, Methods, and Initial Observations.
Elisabeth A Kilroy, Rachael Burris, Edritz Javelosa, Jessica Waits, Angela Lek, Sharon Hesterlee

J Neuromuscul Dis· May 2023

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Gene therapy as a potential therapeutic option for Duchenne muscular dystrophy: A qualitative preference study of patients and parents.
Holly Landrum Peay, Ryan Fischer, Janice P Tzeng, Sharon E Hesterlee, Carl Morris

PLoS One· January 2020

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The Rules of Engagement: CTTI Recommendations for Successful Collaborations Between Sponsors and Patient Groups Around Clinical Trials.
Diane Bloom, Joel Beetsch, Matthew Harker, Sharon Hesterlee, Paulo Moreira

Ther Innov Regul Sci· March 2018

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Assessing the Financial Value of Patient Engagement: A Quantitative Approach from CTTI's Patient Groups and Clinical Trials Project.
Bennett Levitan, Kenneth Getz, Eric L Eisenstein, Michelle Goldberg, Matthew Harker, Sharon Hesterlee

Ther Innov Regul Sci· March 2018

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Patient-Centered Therapy Development for Myotonic Dystrophy: Report of the Myotonic Dystrophy Foundation-Sponsored Workshop.
Sharon Hesterlee, Shashi Amur, Lisa J Bain, John Carulli, Sarah Clarke

Ther Innov Regul Sci· July 2017

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Patient Engagement Practices in Clinical Research among Patient Groups, Industry, and Academia in the United States: A Survey.
Sophia K Smith, Wendy Selig, Matthew Harker, Jamie N Roberts, Sharon Hesterlee

PLoS One· May 2016

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The TREAT-NMD advisory committee for therapeutics (TACT): an innovative de-risking model to foster orphan drug development.
Emma Heslop, Cristina Csimma, Volker Straub, John McCall, Kanneboyina Nagaraju, Sharon Hesterlee

Orphanet J Rare Dis· April 2015

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A call for transparent reporting to optimize the predictive value of preclinical research.
Story C Landis, Susan G Amara, Khusru Asadullah, Chris P Austin, Robi Blumenstein, Sharon E Hesterlee

Nature· October 2012

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Whole-genome analysis of sporadic amyotrophic lateral sclerosis.
Travis Dunckley, Matthew J Huentelman, David W Craig, John V Pearson, Szabolcs Szelinger, Sharon E Hesterlee

N Engl J Med· August 2007

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Challenges in drug development for muscle disease: a stakeholders' meeting.
Jerry R Mendell, Cristina Csimma, Craig M McDonald, Diana M Escolar, Scott Janis, Sharon E Hesterlee

Muscle Nerve· January 2007

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