Search Result "Hurler syndrome"


Mucopolysaccharidosis Type II (Hunter Syndrome): Clinical Picture and Treatment

Journal: Current Pharmaceutical Biotechnology
Volume: 12 Issue: 6 Year: 2011 Page: 861-866
Author(s): Michael Beck

Case Report

Bone Remodeling in an Mps-1h Girl after Hematopoietic Stem CellTransplantation along with Enzymatic Replacement Therapy

Journal: Endocrine, Metabolic & Immune Disorders - Drug Targets
Volume: 22 Issue: 14 Year: 2022 Page: 1425-1432
Author(s): Maria Felicia Faienza

Mucopolysaccharidosis Type III (Sanfilippo Syndrome): Emerging Treatment Strategies

Journal: Current Pharmaceutical Biotechnology
Volume: 12 Issue: 6 Year: 2011 Page: 923-930
Author(s): J. de Ruijter, M. J. Valstar, F. A. Wijburg

Pharmacological Suppression of Premature Stop Mutations that Cause Genetic Diseases

Journal: Current Pharmacogenomics
Volume: 3 Issue: 4 Year: 2005 Page: 259-269
Author(s): K. M. Keeling, D. M. Bedwell

Tetralogy of Fallot and Hypoplastic Left Heart Syndrome – Complex Clinical Phenotypes Meet Complex Genetic Networks

Journal: Current Genomics
Volume: 16 Issue: 3 Year: 2015 Page: 141-158
Author(s): Harald Lahm,Patric Schon,Stefanie Doppler,Martina Dreßen,Julie Cleuziou,Marcus-Andre Deutsch,Peter Ewert,Rudiger Lange,Markus Krane

Intrathecal Enzyme Replacement Therapy for Mucopolysaccharidosis I: Translating Success in Animal Models to Patients

Journal: Current Pharmaceutical Biotechnology
Volume: 12 Issue: 6 Year: 2011 Page: 946-955
Author(s): Patricia I. Dickson, Agnes H. Chen

Cell-and Gene-based Therapeutic Approaches for Neurological Deficits in Mucopolysaccharidoses

Journal: Current Pharmaceutical Biotechnology
Volume: 12 Issue: 6 Year: 2011 Page: 884-896
Author(s): Dao Pan

Cellular Therapy of Lysosomal Storage Disorders: Current Status and Future Prospects

Journal: Current Pediatric Reviews
Volume: 5 Issue: 3 Year: 2009 Page: 147-159
Author(s): Robert F. Wynn, Matthew Stubbs, Nurdan Ozyilmaz, J. Ed Wraith, Brian Bigger

Gene Therapy in Liver Diseases: State-of-the-Art and Future Perspectives

Journal: Current Gene Therapy
Volume: 12 Issue: 6 Year: 2012 Page: 463-483
Author(s): Kalliopi Domvri,Paul Zarogoulidis,Konstantinos Porpodis,Maria Koffa,Maria Lambropoulou,Stylianos Kakolyris,George Kolios,Konstantinos Zarogoulidis,Ekaterini Chatzaki

Role of Nanotechnology for Enzyme Replacement Therapy in Lysosomal Diseases. A Focus on Gaucher’s Disease

Journal: Current Medicinal Chemistry
Volume: 23 Issue: 9 Year: 2016 Page: 929-952
Author(s): L. Martín-Banderas,M.A. Holgado,M. Durán-Lobato,J.J. Infante,J. Álvarez-Fuentes,M. Fernández-Arévalo

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