scholarly journals Long-Term Observation of the Safety and Effectiveness of Enzyme Replacement Therapy in Japanese Patients with Pompe Disease: Results From the Post-marketing Surveillance

2019 ◽  
Vol 8 (2) ◽  
pp. 397-409 ◽  
Author(s):  
Hitoshi Nagura ◽  
Jiro Hokugo ◽  
Kazuo Ueda
Keyword(s):  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Pompe Disease ◽  
Japanese Patients ◽  
Enzyme Replacement ◽  
Post Marketing Surveillance ◽  
Post Marketing ◽  
Long Term Observation

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

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):  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Outcome Measures ◽  
Pompe Disease ◽  
Case Series ◽  
Enzyme Replacement ◽  
Infantile Pompe Disease ◽  
Long Term Survivors

Effect of long term enzyme replacement therapy in late onset Pompe disease: a single-centre experience

2020 ◽  
Author(s):  
George K. Papadimas ◽  
Christoforos Anagnostopoulos ◽  
Sophia Xirou ◽  
Helen Michelakakis ◽  
Gerasimos Terzis ◽  
...  
Keyword(s):  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Pompe Disease ◽  
Late Onset ◽  
Single Centre ◽  
Enzyme Replacement ◽  
Single Centre Experience

scholarly journals Skeletal muscle pathology of infantile Pompe disease during long-term enzyme replacement therapy

2013 ◽  
Vol 8 (1) ◽  
pp. 90 ◽  
Author(s):  
Sean N Prater ◽  
Trusha T Patel ◽  
Anne F Buckley ◽  
Hanna Mandel ◽  
Eugene Vlodavski ◽  
...  
Keyword(s):  
Skeletal Muscle ◽  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Pompe Disease ◽  
Muscle Pathology ◽  
Enzyme Replacement ◽  
Infantile Pompe Disease

scholarly journals Long-term follow-up of 17 patients with childhood Pompe disease treated with enzyme replacement therapy

2018 ◽  
Vol 41 (6) ◽  
pp. 1205-1214 ◽  
Author(s):  
Jan C. van der Meijden ◽  
Michelle E. Kruijshaar ◽  
Laurike Harlaar ◽  
Dimitris Rizopoulos ◽  
Nadine A. M. E. van der Beek ◽  
...  
Keyword(s):  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Pompe Disease ◽  
Enzyme Replacement ◽  
Long Term Follow Up
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