Enzyme replacement therapy in mucopolysaccharidosis type I: Progress and emerging difficulties

Replacement Therapy ◽

Type I ◽

Enzyme replacement therapy with laronidase (Aldurazyme®) for treating mucopolysaccharidosis type I

10.1002/14651858.cd009354.pub2 ◽

2013 ◽

Cited By ~ 11

Author(s):

Elisabeth Jameson ◽

Simon Jones ◽

James E Wraith

Keyword(s):

Replacement Therapy ◽

Type I ◽

Induction of immune tolerance to enzyme replacement therapy in mucopolysaccharidosis type I

10.1016/j.ymgme.2017.12.220 ◽

2018 ◽

Vol 123 (2) ◽

pp. S86

Author(s):

Ai Yin Liao ◽

Arunabha Ghosh ◽

Claire O'Leary ◽

Jean Mercer ◽

Heather J. Church ◽

...

Keyword(s):

Replacement Therapy ◽

Immune Tolerance ◽

Type I ◽

Intra-articular enzyme replacement therapy with rhIDUA is safe, well-tolerated, and reduces articular GAG storage in the canine model of mucopolysaccharidosis type I

10.1016/j.ymgme.2014.05.015 ◽

2014 ◽

Vol 112 (4) ◽

pp. 286-293 ◽

Cited By ~ 8

Author(s):

Raymond Y. Wang ◽

Afshin Aminian ◽

Michael F. McEntee ◽

Shih-Hsin Kan ◽

Calogera M. Simonaro ◽

...

Keyword(s):

Replacement Therapy ◽

Canine Model ◽

Type I ◽

Bull's eye maculopathy and subfoveal deposition in two mucopolysaccharidosis type I patients on long-term enzyme replacement therapy

American Journal of Ophthalmology Case Reports ◽

10.1016/j.ajoc.2017.10.006 ◽

2018 ◽

Vol 9 ◽

pp. 1-6 ◽

Cited By ~ 3

Author(s):

Heather G. Mack ◽

R.C. Andrew Symons ◽

Gerard de Jong

Keyword(s):

Replacement Therapy ◽

Type I ◽

Enzyme Replacement ◽

Bull’S Eye Maculopathy ◽

Bull's Eye

Effects of neonatal enzyme replacement therapy and simvastatin treatment on cervical spine disease in mucopolysaccharidosis type I dogs

10.1016/j.ymgme.2013.12.244 ◽

2014 ◽

Vol 111 (2) ◽

pp. S100

Author(s):

Lachlan J. Smith ◽

Joseph A. Chiaro ◽

Patricia O'Donnell ◽

Neil R. Malhotra ◽

Eileen M. Shore ◽

...

Keyword(s):

Cervical Spine ◽

Replacement Therapy ◽

Type I ◽

Simvastatin Treatment ◽

Enzyme Replacement ◽

Spine Disease

Enzyme replacement therapy with laronidase (Aldurazyme®) for treating mucopolysaccharidosis type I

10.1002/14651858.cd009354 ◽

2011 ◽

Author(s):

Elisabeth Jameson ◽

Simon Jones ◽

James E Wraith

Keyword(s):

Replacement Therapy ◽

Type I ◽

Progressive eye pathology in mucopolysaccharidosis type I mice and effects of enzyme replacement therapy

Clinical and Experimental Ophthalmology ◽

10.1111/ceo.13713 ◽

2020 ◽

Vol 48 (3) ◽

pp. 334-342

Author(s):

Esteban A. Gonzalez ◽

Fernanda Visioli ◽

Gabriela Pasqualim ◽

Carolina F. M. Souza ◽

Diane R. Marinho ◽

...

Keyword(s):

Replacement Therapy ◽

Type I ◽

Efficacy of laronidase therapy in patients with mucopolysaccharidosis type I who initiated enzyme replacement therapy in adult age. A systematic review and meta-analysis

10.1016/j.ymgme.2017.04.004 ◽

2017 ◽

Vol 121 (2) ◽

pp. 138-149 ◽

Cited By ~ 11

Author(s):

Jordi Pérez-López ◽

Montserrat Morales-Conejo ◽

Mónica López-Rodríguez ◽

Álvaro Hermida-Ameijeiras ◽

Marc Moltó-Abad

Keyword(s):

Systematic Review ◽

Replacement Therapy ◽

Meta Analysis ◽

Type I ◽

Adult Age ◽

Comparison of dermatan sulfate and heparan sulfate concentrations in serum, cerebrospinal fluid and urine in patients with mucopolysaccharidosis type I receiving intravenous and intrathecal enzyme replacement therapy

Clinica Chimica Acta ◽

10.1016/j.cca.2020.05.035 ◽

2020 ◽

Vol 508 ◽

pp. 179-184

Author(s):

Haoyue Zhang ◽

Patricia I. Dickson ◽

Ashlee R. Stiles ◽

Agnes H. Chen ◽

Steven Q. Le ◽

...

Keyword(s):

Cerebrospinal Fluid ◽

Heparan Sulfate ◽

Replacement Therapy ◽

Dermatan Sulfate ◽

Type I ◽

Strategies for the Induction of Immune Tolerance to Enzyme Replacement Therapy in Mucopolysaccharidosis Type I

Molecular Therapy — Methods & Clinical Development ◽

10.1016/j.omtm.2019.02.007 ◽

2019 ◽

Vol 13 ◽

pp. 321-333 ◽

Cited By ~ 2

Author(s):

Arunabha Ghosh ◽

Aiyin Liao ◽

Claire O’Leary ◽

Jean Mercer ◽

Karen Tylee ◽

...

Keyword(s):

Replacement Therapy ◽

Immune Tolerance ◽

Type I ◽