Effects of higher and more frequent dosing of alglucosidase alfa and immunomodulation on long‐term clinical outcome of classic infantile Pompe patients

Higher dosing of alglucosidase alfa improves outcomes in children with Pompe disease: a clinical study and review of the literature

(2020) Aleena A. Khan et al.   GENETICS IN MEDICINE

GAA Variants and Phenotypes Among 1079 Patients with Pompe Disease: Data from the Pompe Registry

(2019) Arnold J.J. Reuser et al.   HUMAN MUTATION

Extension of the Pompe mutation database by linking disease‐associated variants to clinical severity

(2019) Monica Y. Niño et al.   HUMAN MUTATION

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

(2018) Esther Poelman et al.   JOURNAL OF PEDIATRICS

Long term clinical history of an Italian cohort of infantile onset Pompe disease treated with enzyme replacement therapy

(2018) Rossella Parini et al.   Orphanet Journal of Rare Diseases

Early higher dosage of alglucosidase alpha in classic Pompe disease

(2018) Marco Spada et al.   JOURNAL OF PEDIATRIC ENDOCRINOLOGY & METABOLISM

Pompe disease in adulthood: effects of antibody formation on enzyme replacement therapy

(2016) Juna M. de Vries et al.   GENETICS IN MEDICINE

Effects of a higher dose of alglucosidase alfa on ventilator-free survival and motor outcome in classic infantile Pompe disease: an open-label single-center study

(2016) C. M. van Gelder et al.   JOURNAL OF INHERITED METABOLIC DISEASE

The role of antibodies in enzyme treatments and therapeutic strategies

(2015) Brian W. Bigger et al.   BEST PRACTICE & RESEARCH CLINICAL ENDOCRINOLOGY & METABOLISM

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

(2015) A. Broomfield et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Enzyme therapy and immune response in relation to CRIM status: the Dutch experience in classic infantile Pompe disease

(2014) Carin M. van Gelder et al.   JOURNAL OF INHERITED METABOLIC DISEASE

B-Cell Depletion and Immunomodulation before Initiation of Enzyme Replacement Therapy Blocks the Immune Response to Acid Alpha-Glucosidase in Infantile-Onset Pompe Disease

(2013) Melissa E. Elder et al.   JOURNAL OF PEDIATRICS

Algorithm for the Early Diagnosis and Treatment of Patients with Cross Reactive Immunologic Material-Negative Classic Infantile Pompe Disease: A Step towards Improving the Efficacy of ERT

(2013) Suhrad G. Banugaria et al.   PLoS One

Successful immune tolerance induction to enzyme replacement therapy in CRIM-negative infantile Pompe disease

(2012) Yoav H. Messinger et al.   GENETICS IN MEDICINE

The impact of antibodies on clinical outcomes in diseases treated with therapeutic protein: Lessons learned from infantile Pompe disease

(2011) Suhrad G. Banugaria et al.   GENETICS IN MEDICINE

High antibody titer in an adult with Pompe disease affects treatment with alglucosidase alfa

(2010) Juna M. de Vries et al.   MOLECULAR GENETICS AND METABOLISM

Cross-reactive immunologic material status affects treatment outcomes in Pompe disease infants

(2009) Priya S. Kishnani et al.   MOLECULAR GENETICS AND METABOLISM

Early Treatment With Alglucosidase Alfa Prolongs Long-Term Survival of Infants With Pompe Disease

(2009) Priya S Kishnani et al.   PEDIATRIC RESEARCH

Pompe's disease

(2008) Ans T van der Ploeg et al.   LANCET