Browsing by Subject rare diseases

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Showing results 1 to 2 of 2
Issue DateTitleAuthor(s)
2012Achieving the "triple aim" for inborn errors of metabolism: a review of challenges to outcomes research and presentation of a new practice-based evidence frameworkPotter, BK; Chakraborty, P; Kronick, JB; Wilson, K; Coyle, D; Feigenbaum, A; Geraghty, MT; Karaceper, MD; Little, J; Mhanni, A; Mitchell, JJ; Siriwardena, K; Wilson, BJ; Syrowatka, A
2020Outcomes in pediatric studies of medium-chain acyl-coA dehydrogenase (MCAD) deficiency and phenylketonuria (PKU): a reviewPugliese, Michael; Tingley, Kylie; Chow, Andrea; Pallone, Nicole; Smith, Maureen; Rahman, Alvi; Chakraborty, Pranesh; Geraghty, Michael T.; Irwin, Julie; Tessier, Laure; Nicholls, Stuart G.; Offringa, Martin; Butcher, Nancy J.; Iverson, Ryan; Clifford, Tammy J.; Stockler, Sylvia; Hutton, Brian; Paik, Karen; Tao, Jessica; Skidmore, Becky; Coyle, Doug; Duddy, Kathleen; Dyack, Sarah; Greenberg, Cheryl R.; Ghai, Shailly Jain; Karp, Natalya; Korngut, Lawrence; Kronick, Jonathan; MacKenzie, Alex; MacKenzie, Jennifer; Maranda, Bruno; Mitchell, John J.; Potter, Murray; Prasad, Chitra; Schulze, Andreas; Sparkes, Rebecca; Taljaard, Monica; Trakadis, Yannis; Walia, Jagdeep; Potter, Beth K.