Establishing core outcome sets for phenylketonuria (PKU) and medium-chain Acyl-CoA dehydrogenase (MCAD) deficiency in children: study protocol for systematic reviews and Delphi surveys

BACKGROUND: Inherited metabolic diseases (IMD) are a large group of rare single-gene disorders that are typically diagnosed early in life. There are important evidence gaps related to the comparative effectiveness of therapies for IMD, which are in part due to challenges in conducting randomized con...

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Veröffentlicht in:Trials
Hauptverfasser: Potter, Beth K., Hutton, Brian, Clifford, Tammy J., Pallone, Nicole, Smith, Maureen, Stockler, Sylvia, Chakraborty, Pranesh, Barbeau, Pauline, Garritty, Chantelle M., Pugliese, Michael, Rahman, Alvi, Skidmore, Becky, Tessier, Laure, Tingley, Kylie, Coyle, Doug, Greenberg, Cheryl R., Korngut, Lawrence, MacKenzie, Alex, Mitchell, John J., Nicholls, Stuart, Offringa, Martin, Schulze, Andreas, Taljaard, Monica
Format: Artigo
Sprache:Inglês
Veröffentlicht: BioMed Central 2017
Schlagworte:
Study Protocol
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5735866/
https://ncbi.nlm.nih.gov/pubmed/29258568
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13063-017-2327-3
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