Selected Publications

Perry B Shieh, Crystal Proud, Tamir Diamond, Catherine A Chapin, Jawad Ahmad, Alan D Salama, Carsten G Bönnemann, Jonathan Soslow, Barry J Byrne, Aravindhan Veerapandiyan, John F Brandsema, Susan Matesanz, Benjamin J Samelson-Jones, Benjamin J Wilkins, Marianne Gerber, Kara E Godwin Wild, Stefanie Mason, Damon Asher, Craig M McDonald, Jerry R Mendell: Learnings from Patient Mortality after Delandistrogene Moxeparvovec Administration: A Report of Two Cases and Expert Committee Considerations for Future Mitigation and Management. Hum Gene Ther 37(1): 262-279, Mar 2026.

Dennis J Keselman, Juliana C Small, Tiffany Seneviratne, Sarah McCague, Taylor L Kaschak, Sabrina W Yum, Andrea O'Brien, John F Brandsema, Tamir Diamond, Kathleen M Loomes, Kimberly Y Lin, Carol A Wittlieb-Weber, Oscar H Mayer, Scott G Daniel, Amy T Waldman, Benjamin J Samelson-Jones, Susan E Matesanz, Lindsey A George : Real-world outcomes of delandistrogene moxeparvovec gene therapy: Motor outcomes and emerging safety concerns Mol Ther 34(1): 123-130, Jan 2026.

Craig M McDonald, Michela Guglieri, Dragana Vučinić, Gyula Acsadi, John F Brandsema, Claudio Bruno, Erika L Finanger, Amy Harper, Mercedes Lopez Lobato, Riccardo Masson, Nuria Muelas, Francina Munell, Yoram Nevo, Yann Péréon, Han Phan, Valeria A Sansone, Mariacristina Scoto, Tracey Willis, Richard S Finkel, Krista Vandenborne, Sara Cazzaniga, Silvia Montrasio, Federica Alessi, Paolo Bettica, Eugenio Mercuri; Givinostat Study 51 Investigators; : Long-Term Evaluation of Givinostat in Duchenne Muscular Dystrophy, and Natural History Comparisons Ann Clin Transl Neurol 12(11): 2335-2348, Nov 2025.

Craig M McDonald, Michela Guglieri, Dragana Vučinić, Gyula Acsadi, John F Brandsema, Claudio Bruno, Erika L Finanger, Amy Harper, Mercedes Lopez Lobato, Riccardo Masson, Nuria Muelas, Francina Munell, Yoram Nevo, Yann Péréon, Han Phan, Valeria A Sansone, Mariacristina Scoto, Tracey Willis, Richard S Finkel, Krista Vandenborne, Sara Cazzaniga, Silvia Montrasio, Federica Alessi, Paolo Bettica, Eugenio Mercuri; Givinostat Study 51 Investigators; Cooperative International Neuromuscular Research Group (CINRG) Duchenne Natural History Study (DNHS) Investigators; ImagingDMD Investigators: Long-Term Evaluation of Givinostat in Duchenne Muscular Dystrophy, and Natural History Comparisons Ann Clin Transl Neurol 11(1): 2335-2348, Nov 2025.

Katlyn Elizabeth McGrattan, Alicia Hofelich Mohr, Anna Miles, Jacqui Allen , Juliet Ochura, Kayla Hernandez, Katie Walsh, Vamshi Rao, Melanie Stevens , Heather McGhee, Keeley Nichols, Morgan Elaine Turksi, Abigail Spoden, Irena Wilson, Mackenzi Coker, Carmen Leon-Astudillo, Leann Schow Smith, John Brandsema, Hiba Farah, Julia Welc, Deborah Levy, Miranda Clements, Tina Duong, Sally Dunaway Young, Graham Schenck, Randal Richardson, Peter Karachunski, Ashley Brown, Allison Brown, Diana Castro, Basil T Darras, Robert J Graham: Differences in swallowing efficacy of disease modifying treatment between infants receiving pre-symptomatic and symptomatic administration Orphanet J Rare Dis 20(1): 526, Oct 2025.

Jodi M Wolff, Nora Capocci, Evrim Atas, Diana X Bharucha-Goebel, John F Brandsema, Russell J Butterfield, Christina B Chadwick, Manuela Corti, Thomas O Crawford, Linda Cripe, John W Day, Tina Duong, Mai K ElMallah, Kevin M Flanigan, Lindsey A George, Natalie L Goedeker, Erica Goude, Sharon Hesterlee, Brian Lin, Natalie K Katz, Susan E Matesanz, Craig McDonald, Elizabeth M McNally, Claudia Mercado-Rodriguez, Deipanjan Nandi, Julie A Parsons, Crystal Proud, Leigh Ramos-Platt, Angela Lek, Stephanie M Salabarria, Eric Camino, Rachel Schrader, Erin Shea, Richard Shell, Perry B Shieh, Jonathan H Soslow, Jane B Taylor, Aravindhan Veerapandiyan, Chet Villa, Michele L Yang, Craig M Zaidman, Carmen Leon-Astudillo, Barry J Byrne: Consensus recommendations and considerations for the delivery and monitoring of gene therapy in patients with Duchenne muscular dystrophy Neuromuscul Disord 54(1): 106208, Sept 2025.

Crystal M Proud, Richard S Finkel, Julie A Parsons, Riccardo Masson, John F Brandsema, Nancy L Kuntz, Richard Foster, Wenjing Li, Ross Littauer, Jihee Sohn, Stephanie Fradette, Bora Youn, Angela D Paradis; RESPOND Study Group: Open-label phase IV trial evaluating nusinersen after onasemnogene abeparvovec in children with spinal muscular atrophy J Clin Invest 16(1): e193956, Sept 2025.

Jiachen Zhou, Sigrid Nilius, Olga Pilipczuk, Anna Scowcroft, Thaïs Tarancón, Frank Tennigkeit, Piotr Zaremba, Nishtha Chandra, Nancy Kuntz, Jonathan Strober, John Brandsema: Treatment Patterns and Disease Burden of Juvenile Myasthenia Gravis in the United States: A Cohort Study Using Health Care Claims Databases Neurology 105(1): e213736, Jul 2025.

Jiachen Zhou, Sigrid Nilius, Olga Pilipczuk, Anna Scowcroft, Thaïs Tarancón, Frank Tennigkeit, Piotr Zaremba, Nishtha Chandra, Nancy Kuntz, Jonathan Strober, John Brandsema : Treatment Patterns and Disease Burden of Juvenile Myasthenia Gravis in the United States: A Cohort Study Using Health Care Claims Databases Neurology 105(1): e213736, Jul 2025.

Dennis J. Keselman & John F. Brandsema: Update on Treatment of Dystrophinopathy. Current Treatment Options in Neurology 27(27), April 2025.