Gene therapy as a potential therapeutic option for Duchenne muscular dystrophy: A qualitative preference study of patients and parents von Landrum Peay, Holly, Fischer, Ryan, Tzeng, Janice P, Hesterlee, Sharon E, Morris, Carl, Strong Martin, Amy, Rensch, Colin, Smith, Edward, Ricotti, Valeria, Beaverson, Katherine, Wand, Hannah, Mansfield, Carol

Volltext

A call for transparent reporting to optimize the predictive value of preclinical research von Landis, Story C., Amara, Susan G., Asadullah, Khusru, Austin, Chris P., Blumenstein, Robi, Bradley, Eileen W., Crystal, Ronald G., Darnell, Robert B., Ferrante, Robert J., Fillit, Howard, Finkelstein, Robert, Fisher, Marc, Gendelman, Howard E., Golub, Robert M., Goudreau, John L., Gross, Robert A., Gubitz, Amelie K., Hesterlee, Sharon E., Howells, David W., Huguenard, John, Kelner, Katrina, Koroshetz, Walter, Krainc, Dimitri, Lazic, Stanley E., Levine, Michael S., Macleod, Malcolm R., McCall, John M., Moxley III, Richard T., Narasimhan, Kalyani, Noble, Linda J., Perrin, Steve, Porter, John D., Steward, Oswald, Unger, Ellis, Utz, Ursula, Silberberg, Shai D.

Volltext

P.34 MDA MOVR clinical data hub provides insights into adoption of approved therapies for neuromuscular disease von Hesterlee, S., Kilroy, E., Waits, J.

Volltext

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

Volltext

Patient Engagement Practices in Clinical Research among Patient Groups, Industry, and Academia in the United States: A Survey von Smith, Sophia K, Selig, Wendy, Harker, Matthew, Roberts, Jamie N, Hesterlee, Sharon, Leventhal, David, Klein, Richard, Patrick-Lake, Bray, Abernethy, Amy P

Volltext

Assessing the Financial Value of Patient Engagement: A Quantitative Approach from CTTI’s Patient Groups and Clinical Trials Project von Levitan, Bennett, Getz, Kenneth, Eisenstein, Eric L., Goldberg, Michelle, Harker, Matthew, Hesterlee, Sharon, Patrick-Lake, Bray, Roberts, Jamie N., DiMasi, Joseph

Volltext

Whole-Genome Analysis of Sporadic Amyotrophic Lateral Sclerosis von Dunckley, Travis, Huentelman, Matthew J, Craig, David W, Pearson, John V, Szelinger, Szabolcs, Joshipura, Keta, Halperin, Rebecca F, Stamper, Chelsea, Jensen, Kendall R, Letizia, David, Hesterlee, Sharon E, Pestronk, Alan, Levine, Todd, Bertorini, Tulio, Graves, Michael C, Mozaffar, Tahseen, Jackson, Carlayne E, Bosch, Peter, McVey, April, Dick, Arthur, Barohn, Richard, Lomen-Hoerth, Catherine, Rosenfeld, Jeffrey, O'Connor, Daniel T, Zhang, Kuixing, Crook, Richard, Ryberg, Henrik, Hutton, Michael, Katz, Jonathan, Simpson, Ericka P, Mitsumoto, Hiroshi, Bowser, Robert, Miller, Robert G, Appel, Stanley H, Stephan, Dietrich A

Volltext

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

Volltext

A Melting Pot of Strategies and Collaboration at the 2025 MDA Conference: Sharon Hesterlee, PhD von Hesterlee, Sharon

Volltext

The Miami Framework for ALS and related neurodegenerative disorders: an integrated view of phenotype and biology von Benatar, Michael, Wuu, Joanne, Huey, Edward D., McMillan, Corey T., Petersen, Ronald C., Postuma, Ronald, McHutchison, Caroline, Dratch, Laynie, Arias, Jalayne J., Crawley, Anita, Houlden, Henry, McDermott, Michael P., Cai, Xueya, Thakur, Neil, Boxer, Adam, Rosen, Howard, Boeve, Bradley F., Dacks, Penny, Cosentino, Stephanie, Abrahams, Sharon, Shneider, Neil, Lingor, Paul, Shefner, Jeremy, Andersen, Peter M., Al-Chalabi, Ammar, Turner, Martin R.

Volltext

Publisher Correction: The Miami Framework for ALS and related neurodegenerative disorders: an integrated view of phenotype and biology von Benatar, Michael, Wuu, Joanne, Huey, Edward D., McMillan, Corey T., Petersen, Ronald C., Postuma, Ronald, McHutchison, Caroline, Dratch, Laynie, Arias, Jalayne J., Crawley, Anita, Houlden, Henry, McDermott, Michael P., Cai, Xueya, Thakur, Neil, Boxer, Adam, Rosen, Howard, Boeve, Bradley F., Dacks, Penny, Cosentino, Stephanie, Abrahams, Sharon, Shneider, Neil, Lingor, Paul, Shefner, Jeremy, Andersen, Peter M., Al-Chalabi, Ammar, Turner, Martin R.

Volltext

The Rules of Engagement: CTTI Recommendations for Successful Collaborations Between Sponsors and Patient Groups Around Clinical Trials von Bloom, Diane, Beetsch, Joel, Harker, Matthew, Hesterlee, Sharon, Moreira, Paulo, Patrick-Lake, Bray, Selig, Wendy, Sherman, Jeffrey, Smith, Sophia K., Valentine, James E., Roberts, Jamie N.

Volltext

New MDA Program for Gene Therapy Development in Ultra-Rare Diseases: Sharon Hesterlee, PhD von Hesterlee, Sharon

Volltext

The myotonic dystrophy experience: a North American cross‐sectional study von Hagerman, Katharine A., Howe, Sarah J., Heatwole, Chad R.

Volltext

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

Volltext

COVID-19 and Muscular Dystrophies, and R&D for Rare Disorders: Sharon Hesterlee, PhD von Hesterlee, Sharon

Volltext

Muscular Dystrophy Association Virtual Conference to Feature Latest Advances in Neuromuscular Therapy and Clinical Care von Hesterlee, Sharon

Volltext

What to Expect from the 2021 MDA Clinical and Scientific Conference: Sharon Hesterlee, PhD von Hesterlee, Sharon

Volltext

Sharon Hesterlee, PhD: Addressing The Challenge of Gathering Data on Rare Diseases von Hesterlee, Sharon

Volltext

Sharon Hesterlee, PhD: How the New MOVR Data Platform Benefits Doctors and Patients von Hesterlee, Sharon

Volltext