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American Journal of Human Genetics|March 4, 2008
Human RFT1 deficiency leads to a disorder of N-linked glycosylationMicha A Haeuptle, François M Pujol, Christine Neupert, et al.
American Journal of Medical Genetics. Part A|June 15, 2011
Residual activity and proteasomal degradation of p.Ser298Pro sulfamidase identified in patients with a mild clinical phenotype of Sanfilippo A syndromeNicole Muschol, Sandra Pohl, Ann Meyer, et al.
Pediatric Nephrology (Berlin, Germany)|February 13, 2007
L1CAM mutation in a boy with hydrocephalus and duplex kidneysMax Christoph Liebau, Andreas Gal, Andrea Superti-Furga, et al.
Journal of Pediatric Rehabilitation Medicine|July 28, 2011
Evaluation of disease severity in mucopolysaccharidosesMichael Beck, Joseph Muenzer, Maurizio Scarpa
Orphanet Journal of Rare Diseases|September 30, 2015
Effectiveness of agalsidase alfa enzyme replacement in Fabry disease: cardiac outcomes after 10 years' treatmentChristoph Kampmann, Amandine Perrin, Michael Beck
Endocrinology, Diabetes & Metabolism Case Reports|September 20, 2017
A novel stop mutation (p.(Gln22*)) of DAX1 (NR0B1) results in late-onset X-linked adrenal hypoplasia congenitaJudith Gerards, Michael M Ritter, Elke Kaminsky, et al.
The Biochemical Journal|September 15, 2005
The forkhead transcription factor Foxi1 directly activates the AE4 promoterIngo Kurth, Moritz Hentschke, Suna Hentschke, et al.
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