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

por 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
Publicado 2019

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

por Peay, Holly L., Fischer, Ryan, Mange, Brennan, Paquin, Ryan S., Smith, Edward C., Sadosky, Alesia, Russo, Leo, Ricotti, Valeria, Rensch, Colin, Morris, Carl, Martin, Amy Strong, Ganot, Annie, Beaverson, Katherine, Mansfield, Carol
Publicado 2021

Priorities when deciding on participation in early-phase gene therapy trials for Duchenne muscular dystrophy: a best–worst scaling experiment in caregivers and adult patients

por Paquin, Ryan S., Fischer, Ryan, Mansfield, Carol, Mange, Brennan, Beaverson, Katherine, Ganot, Annie, Martin, Amy Strong, Morris, Carl, Rensch, Colin, Ricotti, Valeria, Russo, Leo J., Sadosky, Alesia, Smith, Edward C., Peay, Holly L.
Publicado 2019