Electrocardiographic response to enzyme replacement therapy for Pompe disease

Replacement Therapy ◽

Pompe Disease ◽

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

Molecular Genetics and Metabolism ◽

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

Molecular Genetics and Metabolism ◽

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

Molecular Genetics and Metabolism ◽

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 ◽

Response of 33 UK patients with infantile-onset Pompe disease to enzyme replacement therapy

Journal of Inherited Metabolic Disease ◽

10.1007/s10545-015-9898-5 ◽

2015 ◽

Vol 39 (2) ◽

pp. 261-271 ◽

Cited By ~ 31

Author(s):

A. Broomfield ◽

J. Fletcher ◽

J. Davison ◽

N. Finnegan ◽

M. Fenton ◽

...

Keyword(s):

Replacement Therapy ◽

Pompe Disease ◽

Pulmonary outcome measures in long‐term survivors of infantile Pompe disease on enzyme replacement therapy: A case series

Pediatric Pulmonology ◽

10.1002/ppul.24621 ◽

2020 ◽

Vol 55 (3) ◽

pp. 674-681

Author(s):

Mai K. ElMallah ◽

Ankit K. Desai ◽

Erica B. Nading ◽

Stephanie DeArmey ◽

Richard M. Kravitz ◽

...

Keyword(s):

Replacement Therapy ◽

Outcome Measures ◽

Pompe Disease ◽

Case Series ◽

Enzyme Replacement ◽

Infantile Pompe Disease ◽

Long Term Survivors

FIVE-YEARS FOLLOW-UP OF THE RESPIRATORY AND MOTOR INVOLVEMENTS IN PATIENTS WITH LATE ONSET POMPE DISEASE (LOPD) ON IRREGULAR ENZYME REPLACEMENT THERAPY (ERT)

CHEST Journal ◽

10.1016/j.chest.2020.05.415 ◽

2020 ◽

Vol 157 (6) ◽

pp. A371

Author(s):

N. Sousa

Keyword(s):

Replacement Therapy ◽

Pompe Disease ◽

Late Onset ◽