Volume 29; Issue supp-SC

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Clinical Therapeutics
Volume 29; Issue supp-SC

Clinical Therapeutics

Volume 29; Issue supp-SC

1

Foreword

Olaf Bodamer, Patrick B. Deegan

Journal:

Clinical Therapeutics

Year:

2007

Language:

english

File:

PDF, 34 KB

english, 2007

2

Lysosomal Storage Disorders—A Vision of the Future

Journal:

Clinical Therapeutics

Year:

2007

Language:

english

File:

PDF, 42 KB

english, 2007

3

Designing Protein Therapies: Lessons Learned from Cerezyme® for Gaucher Disease

Journal:

Clinical Therapeutics

Year:

2007

Language:

english

File:

PDF, 56 KB

english, 2007

4

Beyond the Primary Biochemical Defect in Type 1 Gaucher Disease

Johannes M.F.G. Aerts, Rolf G. Boot

Journal:

Clinical Therapeutics

Year:

2007

Language:

english

File:

PDF, 56 KB

english, 2007

5

Comprehensive Disease Management Model in Heterogeneous Progressive Disease, Exemplified by Gaucher Disease

Pramod K. Mistry

Journal:

Clinical Therapeutics

Year:

2007

Language:

english

File:

PDF, 55 KB

english, 2007

6

Treatment Goals at Different Stages of Fabry Disease Progression

Christoph Wanner

Journal:

Clinical Therapeutics

Year:

2007

Language:

english

File:

PDF, 55 KB

english, 2007

7

Enzyme Replacement Therapy in Mucopolysaccharidosis Type I: The Sooner the Better

Giovanni V. Coppa, Irene Baldoni, Stefano Bruni, Orazio Gabrielli

Journal:

Clinical Therapeutics

Year:

2007

Language:

english

File:

PDF, 45 KB

english, 2007

8

Successful Pregnancy on Enzyme Replacement Therapy with Cerezyme®

Mirando Mrsić, Ksenija Fumić, Hrvoje Vrčić, Kristina Potočki, Ranka Štern-Padovan, Maja Prutki, Nadira Durakoviæ

Journal:

Clinical Therapeutics

Year:

2007

Language:

english

File:

PDF, 55 KB

english, 2007

9

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., Eugen Mengel, Dr. med.

Journal:

Clinical Therapeutics

Year:

2007

Language:

english

File:

PDF, 70 KB

english, 2007

10

Hematologic and Hemato-Oncologic Aspects of Gaucher Disease

Derralynn Hughes, Maria Domenica Cappellini, Marc Gabriel Berger, Jan Van Droogenbroeck, Maaike de Fost, Dragana Janic, Theodore Marinakis, Hanna Rosenbaum, Jesús Villarubia, Elena Zhukovskaya, Carla

Journal:

Clinical Therapeutics

Year:

2007

Language:

english

File:

PDF, 69 KB

english, 2007

11

The Bone in Gaucher Disease

Neal J. Weinreb

Journal:

Clinical Therapeutics

Year:

2007

Language:

english

File:

PDF, 59 KB

english, 2007

12

Hypertrophic Cardiomyopathy in Anderson-Fabry Disease

Franco Cecchi, Anna Frullini, Iacopo Olivotto, Gabriele Castelli, Mauro Ciaccheri, Fabrizio Martinelli, Francesca Torricelli, Walter Borsini, Francesca Garbini

Journal:

Clinical Therapeutics

Year:

2007

Language:

english

File:

PDF, 59 KB

english, 2007

13

Brain Involvement—The Ultimate Challenge

Journal:

Clinical Therapeutics

Year:

2007

Language:

english

File:

PDF, 48 KB

english, 2007

14

Enzyme Replacement Therapy and Fabry Kidney Disease

David G. Warnock

Journal:

Clinical Therapeutics

Year:

2007

Language:

english

File:

PDF, 64 KB

english, 2007

15

Mucopolysaccharidosis I Patient Narratives

Journal:

Clinical Therapeutics

Year:

2007

Language:

english

File:

PDF, 48 KB

english, 2007

16

Type 1 Nonneuronopathic Gaucher Disease in Children

Journal:

Clinical Therapeutics

Year:

2007

Language:

english

File:

PDF, 58 KB

english, 2007

17

Pompe Disease: A Continuum of Clinical Phenotypes

Marloes Hagemans, Nadine van der Beek, Arnold Reuser, Pieter van Doorn, Ans van der Ploeg

Journal:

Clinical Therapeutics

Year:

2007

Language:

english

File:

PDF, 56 KB

english, 2007

18

Fabry Disease in Pediatrics: The Need for Early Intervention

Frits A. Wijburg

Journal:

Clinical Therapeutics

Year:

2007

Language:

english

File:

PDF, 55 KB

english, 2007

19

The Enhancement of Coping Skills and Compliance to Treatment in Adolescents with a Chronic Serious Disease

Bob F. Last, Martha A. Grootenhuis

Journal:

Clinical Therapeutics

Year:

2007

Language:

english

File:

PDF, 55 KB

english, 2007

20

Longer Survival by Enzyme Replacement Therapy Unmasks the Underrecognition of Otoneurologic Involvement in Infantile-Onset Pompe Disease

Hanna Mandel, Maayan Gruber, Dorit Goldsher, Andrei Chistyakov, Boris Kaplan, Menashe Zaaroor, Hava Hafner

Journal:

Clinical Therapeutics

Year:

2007

Language:

english

File:

PDF, 57 KB

english, 2007

21

A Phase III Extension Study of Aldurazyme®(Laronidase) in Mucopolysaccharidosis I

L.A. Clarke, J.E. Wraith, M. Beck, E.H. Kolodny, G.M. Pastores, J. Muenzer

Journal:

Clinical Therapeutics

Year:

2007

Language:

english

File:

PDF, 51 KB

english, 2007