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Journal Articles

Transformation in Pre-Treatment Presentations of Gaucher Disease during the First Two Decades of Imiglucerase Enzyme Replacement Therapy: A Report from the International Collaborative Gaucher Group Gaucher Registry

Pramod Mistry, MD PhD FRCP, Neal J. Weinreb, MD FACP, Julie Batista, ScM, ScD, Hans C. Andersson, MD, Manisha Balwani, MD MS, Thomas Andrew Burrow, MD, Joel Charrow, MD, Paige Kaplan, MB, BCh, Aneal Khan, MD MSc, FCCMG, FRCPC, Priya S. Kishnani, MD MB BS, Edwin H. Kolodny, MD, Barry E. Rosenbloom, MD, C. Ronald Scott, MD
Journal: Blood
Blood (2016) 128 (22): 4877.
https://doi.org/10.1182/blood.V128.22.4877.4877
Published: 2016
Journal Articles

Lysosomal Newborn Screening in a Cohort in Mexican Population-

Juana Ines Navarrete, MD
Journal: Blood
Blood (2015) 126 (23): 5581.
https://doi.org/10.1182/blood.V126.23.5581.5581
Published: 2015
Journal Articles

Lentiviral gene therapy of murine hematopoietic stem cells ameliorates the Pompe disease phenotype

Niek P. van Til, Merel Stok, Fatima S. F. Aerts Kaya, Monique C. de Waard, Elnaz Farahbakhshian, Trudi P. Visser, Marian A. Kroos, Edwin H. Jacobs, Monique A. Willart, Pascal van der Wegen, Bob J. Scholte, Bart N. Lambrecht, Dirk J. Duncker, Ans T. van der Ploeg, Arnold J. J. Reuser, Monique M. Verstegen, Gerard Wagemaker
Journal: Blood
Blood (2010) 115 (26): 5329–5337.
https://doi.org/10.1182/blood-2009-11-252874
Published: 2010
Includes: Supplemental data