Fostering collaborative research for rare genetic disease: the example of niemann-pick type C disease

by Walkley, Steven U., Davidson, Cristin D., Jacoby, Jonathan, Marella, Philip D., Ottinger, Elizabeth A., Austin, Christopher P., Porter, Forbes D., Vite, Charles H., Ory, Daniel S.
Published 2016

Efficacy and ototoxicity of different cyclodextrins in Niemann–Pick C disease

by Davidson, Cristin D., Fishman, Yonatan I., Puskás, István, Szemán, Julianna, Sohajda, Tamás, McCauliff, Leslie A., Sikora, Jakub, Storch, Judith, Vanier, Marie T., Szente, Lajos, Walkley, Steven U., Dobrenis, Kostantin
Published 2016