Disorders of Protein Misfolding: Alpha-1-Antitrypsin Deficiency as Prototype - 23/07/13
Doi : 10.1016/j.jpeds.2013.03.077
Gary A. Silverman, MD, PhD, Stephen C. Pak, PhD, David H. Perlmutter, MD ⁎ 
Departments of Pediatrics, Cell Biology, and Physiology, University of Pittsburgh School of Medicine, Children's Hospital of Pittsburgh and Magee Women's Hospital of the University of Pittsburgh Medical Center, Pittsburgh, PA
∗Reprint requests: David H. Perlmutter, MD, Department of Pediatrics, University of Pittsburgh School of Medicine, Pittsburgh, PA 15224.
Keyword : AT, ATD, ATM, ATZ, CBZ, COPD, ER, FDA, PBA, RNAi, SNP
Plan
| Funded by the National Institutes of Health (P01DK096990, R01DK084512, R01DK076918, and R01DK079806), Children's Hospital of Pittsburgh, and Magee Women's Hospital of the University of Pittsburgh Medical Center. D.P. is principal investigator of “Carbamazepine for severe liver disease due to alpha-1-antitrypsin deficiency” (clinicaltrials.gov: NCT 01379469). The other authors declare no conflicts of interest. |
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Vol 163 - N° 2
P. 320-326 - août 2013 Retour au numéro
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