Methodology of clinical research in rare diseases: Development of a research program in juvenile neuronal ceroid lipofuscinosis (JNCL) via creation of a patient registry and collaboration with patient advocates
Titel:
Methodology of clinical research in rare diseases: Development of a research program in juvenile neuronal ceroid lipofuscinosis (JNCL) via creation of a patient registry and collaboration with patient advocates
Auteur:
de Blieck, Elisabeth A. Augustine, Erika F. Marshall, Frederick J. Adams, Heather Cialone, Jennifer Dure, Leon Kwon, Jennifer M. Newhouse, Nicole Rose, Katherine Rothberg, Paul G. Vierhile, Amy Mink, Jonathan W.