Assessing the quality and value of metabolic chart data for capturing core outcomes for pediatric medium-chain acyl-CoA dehydrogenase (MCAD) deficiency

• প্রধান লেখক: Ryan Iverson (লেখক), Monica Taljaard (লেখক), Michael T. Geraghty (লেখক), Michael Pugliese (লেখক), Kylie Tingley (লেখক), Doug Coyle (লেখক), Jonathan B. Kronick (লেখক), Kumanan Wilson (লেখক), Valerie Austin (লেখক), Catherine Brunel-Guitton (লেখক), Daniela Buhas (লেখক), Nancy J. Butcher (লেখক), Alicia K. J. Chan (লেখক), Sarah Dyack (লেখক), Sharan Goobie (লেখক), Cheryl R. Greenberg (লেখক), Shailly Jain-Ghai (লেখক), Michal Inbar-Feigenberg (লেখক), Natalya Karp (লেখক), Mariya Kozenko (লেখক), Erica Langley (লেখক), Matthew Lines (লেখক), Julian Little (লেখক), Jennifer MacKenzie (লেখক), Bruno Maranda (লেখক), Saadet Mercimek-Andrews (লেখক), Aizeddin Mhanni (লেখক), John J. Mitchell (লেখক), Laura Nagy (লেখক), Martin Offringa (লেখক), Amy Pender (লেখক), Murray Potter (লেখক), Chitra Prasad (লেখক), Suzanne Ratko (লেখক), Ramona Salvarinova (লেখক), Andreas Schulze (লেখক), Komudi Siriwardena (লেখক), Neal Sondheimer (লেখক), Rebecca Sparkes (লেখক), Sylvia Stockler-Ipsiroglu (লেখক), Kendra Tapscott (লেখক), Yannis Trakadis (লেখক), Lesley Turner (লেখক), Clara Van Karnebeek (লেখক), Anthony Vandersteen (লেখক), Jagdeep S. Walia (লেখক), Brenda J. Wilson (লেখক), Andrea C. Yu (লেখক), Beth K. Potter (লেখক), Pranesh Chakraborty (লেখক)
• বিন্যাস: গ্রন্থ
• প্রকাশিত: BMC, 2024-01-01T00:00:00Z.
• বিষয়গুলি: article
• অনলাইন ব্যবহার করুন: Connect to this object online.

Abstract Background Generating rigorous evidence to inform care for rare diseases requires reliable, sustainable, and longitudinal measurement of priority outcomes. Having developed a core outcome set for pediatric medium-chain acyl-CoA dehydrogenase (MCAD) deficiency, we aimed to assess the feasibi...