Table of Contents

Preface pp. i -xiv

Chapter 1. Sialidosis: Pathophysiology and Therapeutic Approaches
(Eugen-Matthias Strehle) pp.1-14

Chapter 2. Gaucher disease and therapeutic approaches
(Deborah Elstein, Ehud Lebel, Gheona Altarescu, Irith Hadas-Halpern and Ari Zimran)pp.15-36

Chapter 3. Parkinson’s disease: molecular changes and therapeutic approaches
(Erich Richter, Marina Abramova and Sankar Surendran)pp.37-56

Chapter 4. Ataxia-telangiectasia: Clinical symptoms and therapeutic approaches
(Aggeliki Kolialexi and Ariadni Mavrou)pp.57-70

Chapter 5. Rett syndrome and therapeutic approaches
(Karine Pelc, Guy Cheron and Bernard Dan)pp.71-82

Chapter 6. Krabbe Disease and therapeutic approaches
(Patricia K. Duffner)pp.83-108

Chapter 7. Gangliosidoses: molecular pathology and therapeutic approaches
(Yoshiyuki Suzuki)pp.109-128

Chapter 8. Enzyme replacement therapy for lysosomal storage diseases
(Shunji Tomatsu,, Adriana M. Montaño, Angela Catalina Sosa Molano,et al.) pp.129-158

Chapter 9. Cell-based Gene Therapy for Lysosomal Storage Diseases
(Seung U. Kim)pp.159-168

Chapter 10. Biomarkers for lysosomal storage disorders
(Johannes M. F. G. Aerts, Maria Joao Ferraz, Rolf G. Boot, et al.)pp.169-202

Chapter 11. PKU in Turkey: screening, diagnosis and management
(Hülya Gökmen-Özel and Turgay Coþkun)pp.203-218

Chapter 12. Phenylketonuria and therapeutic approaches
(Erin L. MacLeod and Denise M. Ney)pp.219-240

Chapter 13. Canavan disease: molecular pathology, phenotype and therapeutic approaches
(Sankar Surendran, Namik Kaya and Pinar Ozand)pp.241-264

Contributors pp.265-268

Index pp.269-287