Volume 29, Supplement C, 2007
Selected Proceedings of the 4th Symposium on Lysosomal Storage Disorders
Olaf Bodamer, MD, PhD, FACMG; and
Patrick B. Deegan, MD, MRCPI, MRCPath
Guest Editors
Contents
Foreword
Olaf Bodamer, MD, PhD, FACMG; and Patrick B. Deegan, MD, MRCPI, MRCPath
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Lysosomal Storage Disorders—A Vision of the Future
Alan Smith, PhD
[PDF]
Designing Protein Therapies: Lessons Learned from Cerezyme® for Gaucher Disease
Tim Edmunds, PhD
[PDF]
Beyond the Primary Biochemical Defect in Type 1 Gaucher Disease
Johannes M.F.G. Aerts, PhD; and Rolf G. Boot, PhD
[PDF]
Comprehensive Disease Management Model in Heterogeneous Progressive Disease, Exemplified by Gaucher Disease
Pramod K. Mistry, MD, PhD, FRCP
[PDF]
Treatment Goals at Different Stages of Fabry Disease Progression
Christoph Wanner, MD
[PDF]
Enzyme Replacement Therapy in Mucopolysaccharidosis Type I: The Sooner the Better
Giovanni V. Coppa, MD; Irene Baldoni, MD; Stefano Bruni, MD; and Orazio Gabrielli, MD
[PDF]
Successful Pregnancy on Enzyme Replacement Therapy with Cerezyme®
Mirano Mrsic, MD, PhD; Ksenija Fumic, PhD; Hrvoje Vrcic, MD, PhD;
Kristina Potocki, MD, PhD; Ranka Stern-Padovan, MD, PhD; Maja Prutki, MD;
and Nadira Durakoviæ, MD
[PDF]
Initial Therapy Response of 6 Months of Enzyme Replacement Therapy in 11 Juvenile/Adult M. Pompe Patients
Ralf Hartung; Fares Chamsi-Bacha; Michael Beck, Prof. Dr. med.; and Eugen Mengel, Dr. med.
[PDF]
Hematologic and Hemato-Oncologic Aspects of Gaucher Disease
Derralynn Hughes, MA, DPhil, MRCP, MRCPath; Maria Domenica Cappellini, MD; Marc Gabriel Berger, MD, PhD; Jan Van Droogenbroeck, MD; Maaike de Fost, MD; Dragana Janic, MD, PhD; Theodore Marinakis, MD, PhD; Hanna Rosenbaum, MD; Jesús Villarubia, MD, PhD; Elena Zhukovskaya, MD; and Carla Hollak, MD
[PDF]
The Bone in Gaucher Disease
Neal J. Weinreb, MD, FACP
[PDF]
Hypertrophic Cardiomyopathy in Anderson-Fabry Disease
Franco Cecchi; Anna Frullini; Iacopo Olivotto; Gabriele Castelli; Mauro Ciaccheri; Fabrizio Martinelli; Francesca Torricelli; Walter Borsini; and Francesca Garbini
[PDF]
Brain Involvement—The Ultimate Challenge
Eugen Mengel, Dr. med.
[PDF]
Enzyme Replacement Therapy and Fabry Kidney Disease
David G. Warnock, MD
[PDF]
Mucopolysaccharidosis I Patient Narratives
Rachel Lawes, PhD
[PDF]
Type 1 Nonneuronopathic Gaucher Disease in Children
Paige Kaplan, MBBCh
[PDF]
Pompe Disease: A Continuum of Clinical Phenotypes
Marloes Hagemans, PhD; Nadine van der Beek, MD; Arnold Reuser, PhD; Pieter van Doorn, MD, PhD; and Ans van der Ploeg, MD, PhD
[PDF]
Fabry Disease in Pediatrics: The Need for Early Intervention
Frits A. Wijburg, MD, PhD
[PDF]
The Enhancement of Coping Skills and Compliance to Treatment in Adolescents with a Chronic Serious Disease
Bob F. Last, PhD; and Martha A. Grootenhuis, PhD
[PDF]
Longer Survival by Enzyme Replacement Therapy Unmasks the Underrecognition of Otoneurologic Involvement in Infantile-Onset Pompe Disease
Hanna Mandel, MD; Maayan Gruber, MD; Dorit Goldsher, MD; Andrei Chistyakov, PhD; Boris Kaplan, MSc; Menashe Zaaroor, MD, PhD; and Hava Hafner, PhD
[PDF]
A Phase III Extension Study of Aldurazyme® (Laronidase) in Mucopolysaccharidosis I
L.A. Clarke, MD; J.E. Wraith; M. Beck, MD; E.H. Kolodny, MD; G.M. Pastores; and J. Muenzer
[PDF]
Poster Abstracts
[PDF]
Support for this supplement was provided by Genzyme Europe BV, Naarden, The Netherlands. The opinions are those of the authors and not necessarily those of the sponsor.
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