搜索页_ teresa.buracchio@fda.hhs.gov class= auth_mail title= E-mail the corresponding author

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1.Neurocognitive clinical outcome assessments for inborn errors of metabolism and other rare conditions

作者：Elsa Shapiro^a ; ^b ; ^{shapi004@umn.edu" class="auth_mail" title="E-mail the corresponding author} ; Jessica Bernstein^c ; ^{jessica.bernstein@fda.hhs.gov" class="auth_mail" title="E-mail the corresponding author} ; Heather R. Adams^d ; ^{Heather_Adams@URMC.Rochester.edu" class="auth_mail" title="E-mail the corresponding author} ; Ann J. Barbier^e ; ¹ ; ^{annjbarbier@aol.com" class="auth_mail" title="E-mail the corresponding author} ; Teresa Buracchio^c ; ^{teresa.buracchio@fda.hhs.gov" class="auth_mail" title="E-mail the corresponding author} ; Peter Como^f ; ^{peter.como@fda.hhs.gov" class="auth_mail" title="E-mail the corresponding author} ; Kathleen A. Delaney^b ; ^a ; ^{kate.delaney@shapiroanddelaney.com" class="auth_mail" title="E-mail the corresponding author} ; Florian Eichler^g ; ^{feichler@partners.org" class="auth_mail" title="E-mail the corresponding author} ; Jonathan C. Goldsmith^c ; ^{jonathan.goldsmith@fda.hhs.gov" class="auth_mail" title="E-mail the corresponding author} ; Melissa Hogan^h ; ^{melissa@savingcase.com" class="auth_mail" title="E-mail the corresponding author} ; Sarrit Kovacs^c ; ^{sarrit.kovacs@fda.hhs.gov" class="auth_mail" title="E-mail the corresponding author} ; Jonathan W. Minkⁱ ; ^{Jonathan_Mink@URMC.Rochester.edu" class="auth_mail" title="E-mail the corresponding author} ; Joanne Odenkirchen^j ; ^{jo21x@nih.gov" class="auth_mail" title="E-mail the corresponding author} ; Melissa A. Parisi^k ; ^{parisima@mail.nih.gov" class="auth_mail" title="E-mail the corresponding author} ; Alison Skrinar^l ; ^{ASkrinar@ultragenyx.com" class="auth_mail" title="E-mail the corresponding author} ; Susan E. Waisbren^m ; ^{Susan.Waisbren@childrens.harvard.edu" class="auth_mail" title="E-mail the corresponding author} ; Andrew E. Mulberg^c ; ^{andrew.mulberg@fda.hhs.gov" class="auth_mail" title="E-mail the corresponding author}

关键词：Inborn errors ; Neurocognitive ; Clinical outcome assessment ; Natural history studies ; Rare diseases ; Drug development

刊名：Molecular Genetics and Metabolism

出版年：2016

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