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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

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

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

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

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

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

Chloramphenicol Enhances IDUA Activity on Fibroblasts from Mucopolysaccharidosis I Patients

Journal: Current Pharmaceutical Biotechnology

Volume: 14 Issue: 2 Year: 2013 Page: 194-198

Author(s): Fabiana Quoos Mayer,Osvaldo Alfonso Artigalas,Valeska Lizzi Lagranha,Guilherme Baldo,Ida Vanessa Schwartz,Ursula Matte,Roberto Giugliani

Review Article
Treatment for Lysosomal Storage Disorders

Journal: Current Pharmaceutical Design

Volume: 26 Issue: 40 Year: 2020 Page: 5110-5118

Author(s): Jayesh Sheth,Aadhira Nair

Mesenchymal Stem Cells for Treatment of CNS Injury

Journal: Current Neuropharmacology

Volume: 8 Issue: 4 Year: 2010 Page: 316-323

Author(s): Michael F. Azari, Louisa Mathias, Ezgi Ozturk, David S. Cram, Richard L. Boyd, Steven Petratos

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