Development of high sustained IgG antibody titers and corresponding clinical decline in an adolescent with atypical infantile Pompe disease after 11+ years on enzyme replacement therapy with alglucosidase alfa

2020 ◽  
Vol 129 (2) ◽  
pp. S89
Author(s):  
Katherine H. Kim ◽  
Erika Vucko ◽  
Ankit K. Desai ◽  
Priya Kishnani ◽  
Barbara K. Burton
Keyword(s):  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Pompe Disease ◽  
Igg Antibody ◽  
Enzyme Replacement ◽  
Alglucosidase Alfa ◽  
Antibody Titers ◽  
Infantile Pompe Disease

scholarly journals High Sustained Antibody Titers in Patients with Classic Infantile Pompe Disease Following Immunomodulation at Start of Enzyme Replacement Therapy

2018 ◽  
Vol 195 ◽  
pp. 236-243.e3 ◽  
Author(s):  
Esther Poelman ◽  
Marianne Hoogeveen-Westerveld ◽  
Marian A. Kroos-de Haan ◽  
Johanna M.P. van den Hout ◽  
Kees J. Bronsema ◽  
...  
Keyword(s):  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Pompe Disease ◽  
Enzyme Replacement ◽  
Antibody Titers ◽  
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 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

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
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