Experiences of patients with lysosomal storage disorders treated with enzyme replacement therapy

Replacement Therapy ◽

Lysosomal Storage ◽

Enzyme Replacement ◽

The use of port-a-caths in adult patients with Lysosomal Storage Disorders receiving Enzyme Replacement Therapy-one centre experience

Molecular Genetics and Metabolism Reports ◽

10.1016/j.ymgmr.2017.10.003 ◽

2017 ◽

Vol 13 ◽

pp. 111-114 ◽

Cited By ~ 3

Author(s):

Mairead McLoughlin ◽

Karolina M. Stepien ◽

Briony McNelly ◽

Lorraine Thompson ◽

Janet Gorton ◽

...

Keyword(s):

Replacement Therapy ◽

Adult Patients ◽

Lysosomal Storage ◽

Enzyme Replacement ◽

The NCS-LSD cohort study: a description of the methods and analyses used to assess the long-term effectiveness of enzyme replacement therapy and substrate reduction therapy in patients with lysosomal storage disorders

Journal of Inherited Metabolic Disease ◽

10.1007/s10545-014-9679-6 ◽

2014 ◽

Vol 37 (6) ◽

pp. 939-944 ◽

Cited By ~ 8

Author(s):

W. E. Henley ◽

L. J. Anderson ◽

K. M. Wyatt ◽

V. Nikolaou ◽

R. Anderson ◽

...

Keyword(s):

Cohort Study ◽

Replacement Therapy ◽

Substrate Reduction Therapy ◽

Lysosomal Storage ◽

Enzyme Replacement ◽

Substrate Reduction ◽

132. Home enzyme replacement therapy for paediatric lysosomal storage disorders: The Manchester experience

Molecular Genetics and Metabolism ◽

10.1016/j.ymgme.2008.11.133 ◽

2009 ◽

Vol 96 (2) ◽

pp. S41

Author(s):

Jones Simon ◽

S. Bagewadi ◽

B.J. Mercer ◽

G. Moss ◽

J. Roberts ◽

...

Keyword(s):

Replacement Therapy ◽

Lysosomal Storage ◽

Enzyme Replacement ◽

The impact of the immune system on the safety and efficiency of enzyme replacement therapy in lysosomal storage disorders

Journal of Inherited Metabolic Disease ◽

10.1007/s10545-016-9917-1 ◽

2016 ◽

Vol 39 (4) ◽

pp. 499-512 ◽

Cited By ~ 10

Author(s):

A. Broomfield ◽

S. A. Jones ◽

S. M. Hughes ◽

B. W. Bigger

Keyword(s):

Immune System ◽

Replacement Therapy ◽

Lysosomal Storage ◽

Enzyme Replacement ◽

The Impact ◽

Enzyme Replacement Therapy for Lysosomal Storage Disorders

Recent Patents on Biomedical Engineering ◽

10.2174/1874764710801020141 ◽

2008 ◽

Vol 1 (2) ◽

pp. 141-147 ◽

Cited By ~ 1

Author(s):

Daniel Sayut ◽

Pavan Kambam ◽

Lianhong Sun

Keyword(s):

Replacement Therapy ◽

Lysosomal Storage ◽

Enzyme Replacement ◽

Lysosomal Storage Disorders and Enzyme Replacement Therapy

Pharmaceutical Biocatalysis ◽

10.1201/9781003045397-11 ◽

2020 ◽

pp. 347-438

Author(s):

Peter Grunwald

Keyword(s):

Replacement Therapy ◽

Lysosomal Storage ◽

Enzyme Replacement ◽

142. A systematic approach towards identifying and treating delayed infusion reactions to enzyme replacement therapy in patients with lysosomal storage disorders

Molecular Genetics and Metabolism ◽

10.1016/j.ymgme.2008.11.143 ◽

2009 ◽

Vol 96 (2) ◽

pp. S43

Author(s):

Jeanine Utz ◽

Brenda Diethelm-Okita ◽

Chester B. Whitley

Keyword(s):

Replacement Therapy ◽

Systematic Approach ◽

Lysosomal Storage ◽

Enzyme Replacement ◽

Infusion Reactions ◽

Enzyme replacement therapy for lysosomal storage disorders in India

Molecular Cytogenetics ◽

10.1186/1755-8166-7-s1-i29 ◽

2014 ◽

Vol 7 (Suppl 1) ◽

pp. I29

Author(s):

Mamta Muranjan

Keyword(s):

Replacement Therapy ◽

Lysosomal Storage ◽

Enzyme Replacement ◽

The COVID-19 Pandemic and Enzyme Replacement Therapy in Lysosomal Storage Disorders

The Journal of Pediatric Research ◽

10.4274/jpr.galenos.2020.98250 ◽

2021 ◽

Vol 0 (0) ◽

pp. 370-376

Author(s):

Asburçe Olgaç ◽

Çiğdem Seher Kasapkara ◽

Burak Açıkel ◽

Yılmaz Yıldız ◽

Gülhan Karakaya Molla ◽

...

Keyword(s):

Replacement Therapy ◽

Lysosomal Storage ◽

Enzyme Replacement ◽

Development of New Strategies for Enzyme Replacement Therapy for Lysosomal Storage Disorders

Journal of mucopolysaccharidosis and rare disease ◽

10.19125/jmrd.2016.2.1.17 ◽

2016 ◽

Vol 2 (1) ◽

pp. 17-18

Author(s):

Ah-Ra Ko

Keyword(s):

Replacement Therapy ◽

Lysosomal Storage ◽

Enzyme Replacement ◽

New Strategies ◽