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2. Patients’ and caregivers’ maximum acceptable risk of death for non‐curative gene therapy to treat Duchenne muscular dystrophy

Author

Peay, Holly L., primary, Fischer, Ryan, additional, Mange, Brennan, additional, Paquin, Ryan S., additional, Smith, Edward C., additional, Sadosky, Alesia, additional, Russo, Leo, additional, Ricotti, Valeria, additional, Rensch, Colin, additional, Morris, Carl, additional, Martin, Amy Strong, additional, Ganot, Annie, additional, Beaverson, Katherine, additional, and Mansfield, Carol, additional

Published
2021
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3. Gene therapy as a potential therapeutic option for Duchenne muscular dystrophy: A qualitative preference study of patients and parents

Author

Landrum Peay, Holly, primary, Fischer, Ryan, additional, Tzeng, Janice P., additional, Hesterlee, Sharon E., additional, Morris, Carl, additional, Strong Martin, Amy, additional, Rensch, Colin, additional, Smith, Edward, additional, Ricotti, Valeria, additional, Beaverson, Katherine, additional, Wand, Hannah, additional, and Mansfield, Carol, additional

Published
2019
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