Long‐term benefit of enzyme replacement therapy with alglucosidase alfa in adults with Pompe disease: Prospective analysis from the French Pompe Registry

2020 ◽  
Vol 43 (6) ◽  
pp. 1219-1231 ◽  
Author(s):  
Claudio Semplicini ◽  
Marie De Antonio ◽  
Nadjib Taouagh ◽  
Anthony Béhin ◽  
Françoise Bouhour ◽  
...  
Keyword(s):  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Pompe Disease ◽  
Prospective Analysis ◽  
Enzyme Replacement ◽  
Alglucosidase Alfa ◽  
Term Benefit

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

scholarly journals Enzyme replacement therapy with alglucosidase alfa in Pompe disease: Clinical experience with rate escalation

2018 ◽  
Vol 123 (2) ◽  
pp. 92-96 ◽  
Author(s):  
Ankit K. Desai ◽  
Crista K. Walters ◽  
Heidi L. Cope ◽  
Zoheb B. Kazi ◽  
Stephanie M. DeArmey ◽  
...  
Keyword(s):  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Clinical Experience ◽  
Pompe Disease ◽  
Enzyme Replacement ◽  
Alglucosidase Alfa

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
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