Defining Natural History: Assessment of the Ability of College Students to Aid in Characterizing Clinical Progression of Niemann-Pick Disease, Type C

Niemann-Pick Disease, type C (NPC) is a fatal, neurodegenerative, lysosomal storage disorder. It is a rare disease with broad phenotypic spectrum and variable age of onset. These issues make it difficult to develop a universally accepted clinical outcome measure to assess urgently needed therapies....

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Detalles Bibliográficos
Autores principales: Shin, Jenny, Epperson, Katrina, Yanjanin, Nicole M., Albus, Jennifer, Borgenheimer, Laura, Bott, Natalie, Brennan, Erin, Castellanos, Daniel, Cheng, Melissa, Clark, Michael, Devany, Margaret, Ensslin, Courtney, Farivari, Nina, Fernando, Shanik, Gabriel, Lauren, Gallardo, Rani, Castleman, Moriah, Gutierrez, Olimpia, Herschel, Allison, Hodge, Sarah, Horst, Anne, Howard, Mary, James, Evan, Jones, Lindsey, Kearns, Mary, Kelly, Mary, Kim, Christine, Kiser, Kinzie, Klazura, Gregory, Knoedler, Chris, Kolbus, Emily, Lange, Lauren, Lee, Joan, Li, Eileena, Lu, Wei, Luttrell, Andrew, Ly, Emily, McKeough, Katherine, McSorley, Brianna, Miller, Catherine, Mitchell, Sean, Moon, Abbey, Moser, Kevin, O'Brien, Shane, Olivieri, Paula, Patzwahl, Aaron, Pereira, Marie, Pymento, Craig, Ramelb, Erin, Ramos, Bryce, Raya, Teresa, Riney, Stephen, Roberts, Geoff, Robertshaw, Mark, Rudolf, Frannie, Rund, Samuel, Sansone, Stephanie, Schwartz, Lindsay, Shay, Ryan, Siu, Edwin, Spear, Timothy, Tan, Catherine, Truong, Marisa, Uddin, Mairaj, VanTrieste, Jennifer, Veloz, Omar, White, Elizabeth, Porter, Forbes D., Haldar, Kasturi
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2011
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3184943/
https://www.ncbi.nlm.nih.gov/pubmed/21984891
http://dx.doi.org/10.1371/journal.pone.0023666

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