P3.53 Quantitative muscle MRI findings in three patients with adult-onset Pompe disease after a 24-month enzyme replacement therapy

Replacement Therapy ◽

Pompe Disease ◽

Adult Onset ◽

Mri Findings ◽

Muscle Mri ◽

Observational clinical study of 22 adult-onset Pompe disease patients undergoing enzyme replacement therapy over 5years

10.1016/j.ymgme.2016.01.013 ◽

2016 ◽

Vol 117 (4) ◽

pp. 413-418 ◽

Cited By ~ 24

Author(s):

Karolina M. Stepien ◽

Christian J. Hendriksz ◽

Mark Roberts ◽

Reena Sharma

Keyword(s):

Clinical Study ◽

Replacement Therapy ◽

Pompe Disease ◽

Adult Onset ◽

Enzyme replacement therapy in adult-onset glycogenosis II: Is quantitative muscle MRI helpful?

Muscle & Nerve ◽

10.1002/mus.21304 ◽

2009 ◽

Vol 40 (1) ◽

pp. 122-125 ◽

Cited By ~ 20

Author(s):

A. Pichiecchio ◽

G.U. Poloni ◽

S. Ravaglia ◽

M. Ponzio ◽

G. Germani ◽

...

Keyword(s):

Replacement Therapy ◽

Adult Onset ◽

Muscle Mri ◽

Enzyme Replacement ◽

Glycogenosis Ii

Cardiac and motor improvement with enzyme replacement therapy in a patient with infantile-onset Pompe disease

Neuropediatrics ◽

10.1055/s-2005-867997 ◽

2005 ◽

Vol 36 (02) ◽

Author(s):

M Smitka ◽

M von der Hagen ◽

A Kaindl ◽

C Gilitzer ◽

J Dumontier ◽

...

Keyword(s):

Replacement Therapy ◽

Pompe Disease ◽

Enzyme Replacement ◽

Motor Improvement

Description of a patient with infantile onset Pompe disease after 45 months of enzyme replacement therapy

10.1016/j.ymgme.2017.12.339 ◽

2018 ◽

Vol 123 (2) ◽

pp. S124-S125

Author(s):

Roberto Sandobal Pacheco ◽

Diana Espinosa Villanueva ◽

Adriana Alcnatara Salinas ◽

Jorge A. Romero Ramirez ◽

Jose Antonio Vasquez Galeana ◽

...

Keyword(s):

Replacement Therapy ◽

Pompe Disease ◽

The effectiveness of enzyme replacement therapy for juvenile‐onset Pompe disease: A systematic review

Journal of Inherited Metabolic Disease ◽

10.1002/jimd.12027 ◽

2019 ◽

Vol 42 (1) ◽

pp. 57-65 ◽

Cited By ~ 6

Author(s):

Joanne Milverton ◽

Skye Newton ◽

Tracy Merlin

Keyword(s):

Systematic Review ◽

Replacement Therapy ◽

Pompe Disease ◽

Juvenile Onset ◽

A 4-year follow-up study of 24 patients with late onset Pompe disease treated with alglucosidase alfa enzyme replacement therapy at a single centre

10.1016/j.ymgme.2014.12.252 ◽

2015 ◽

Vol 114 (2) ◽

pp. S111

Author(s):

Karolina M. Stepien ◽

Jane Whitby ◽

Mark Roberts ◽

Reena Sharma

Keyword(s):

Replacement Therapy ◽

Pompe Disease ◽

Late Onset ◽

Single Centre ◽

Enzyme Replacement ◽

Follow Up Study ◽

Alglucosidase Alfa

Proteomics to identify signature proteins in patients likely to mount an immune response to enzyme replacement therapy in infantile Pompe disease

10.1016/j.ymgme.2014.12.128 ◽

2015 ◽

Vol 114 (2) ◽

pp. S61

Author(s):

Zoheb Kazi ◽

Katie Berrier ◽

Cheng Lu ◽

Deeksha Bali ◽

J. Will Thompson ◽

...

Keyword(s):

Immune Response ◽

Replacement Therapy ◽

Pompe Disease ◽

Enzyme Replacement ◽

Infantile Pompe Disease

Reduced Efficacy of Enzyme Replacement Therapy in a Child with Late-Onset Pompe Disease

Pediatrics & Therapeutics ◽

10.4172/2161-0665.1000290 ◽

2016 ◽

Vol 06 (02) ◽

Cited By ~ 1

Author(s):

Satoru Takahashi ◽

Ryosuke Tanaka

Keyword(s):

Replacement Therapy ◽

Pompe Disease ◽

Late Onset ◽

Improved Exercise Tolerance after Enzyme Replacement Therapy in Pompe Disease

Medicine & Science in Sports & Exercise ◽

10.1249/mss.0b013e31823e6579 ◽

2012 ◽

Vol 44 (5) ◽

pp. 771-775 ◽

Cited By ~ 9

Author(s):

MAURO MARZORATI ◽

SIMONE PORCELLI ◽

BARBARA REGGIORI ◽

LUCIA MORANDI ◽

BRUNO GRASSI

Keyword(s):

Replacement Therapy ◽

Pompe Disease ◽

Exercise Tolerance ◽

Electrocardiographic response to enzyme replacement therapy for Pompe disease

Genetics in Medicine ◽

10.1097/01.gim.0000195896.04069.5f ◽

2006 ◽

Vol 8 (5) ◽

pp. 297-301 ◽

Cited By ~ 41

Author(s):

Annette K Ansong ◽

Jennifer S Li ◽

Eva Nozik-Grayck ◽

Richard Ing ◽

Richard M Kravitz ◽

...

Keyword(s):

Replacement Therapy ◽

Pompe Disease ◽