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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Gepubliceerd in:Trials
Hoofdauteurs: 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
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: BioMed Central 2017
Onderwerpen:
Study Protocol
Online toegang: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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