scholarly journals Characterization of immune response in Cross-Reactive Immunological Material (CRIM)-positive infantile Pompe disease patients treated with enzyme replacement therapy

2019 ◽  
Vol 20 ◽  
pp. 100475 ◽  
Author(s):  
Ankit K. Desai ◽  
Zoheb B. Kazi ◽  
Deeksha S. Bali ◽  
Priya S. Kishnani
Keyword(s):  
Immune Response ◽  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Pompe Disease ◽  
Enzyme Replacement ◽  
Infantile Pompe Disease

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 Motor Development as a Potential Marker to Monitor Infantile Pompe Disease on Enzyme Replacement Therapy

2017 ◽  
Vol 07 (01) ◽  
pp. 8-19 ◽  
Author(s):  
Paula de Almeida Thomazinho ◽  
Fernanda Bertão Scalco ◽  
Maria Lúcia Costa de Oliveira ◽  
Dafne Dain Gandelman Horovitz ◽  
Juan Clinton Llerena Jr.
Keyword(s):  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Motor Development ◽  
Pompe Disease ◽  
Enzyme Replacement ◽  
Potential Marker ◽  
Infantile Pompe Disease

scholarly journals Ocular and Histologic Findings in a Series of Children With Infantile Pompe Disease Treated With Enzyme Replacement Therapy

2014 ◽  
Vol 51 (6) ◽  
pp. 355-362 ◽  
Author(s):  
S. Grace Prakalapakorn ◽  
Alan D. Proia ◽  
Tammy L. Yanovitch ◽  
Stephanie DeArmey ◽  
Nancy J. Mendelsohn ◽  
...  
Keyword(s):  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Pompe Disease ◽  
Enzyme Replacement ◽  
Infantile Pompe Disease

scholarly journals Characterization of pre- and post-treatment pathology after enzyme replacement therapy for pompe disease

2006 ◽  
Vol 86 (12) ◽  
pp. 1208-1220 ◽  
Author(s):  
Beth L Thurberg ◽  
Colleen Lynch Maloney ◽  
Charles Vaccaro ◽  
Kendra Afonso ◽  
Anne Chun-Hui Tsai ◽  
...  
Keyword(s):  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Pompe Disease ◽  
Post Treatment ◽  
Enzyme Replacement
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