Efficacy, safety profile, and immunogenicity of alglucosidase alfa produced at the 4,000-liter scale in US children and adolescents with Pompe disease: ADVANCE, a phase IV, open-label, prospective study

Very Early Treatment for Infantile-Onset Pompe Disease Contributes to Better Outcomes

(2016) Chia-Feng Yang et al.   JOURNAL OF PEDIATRICS

Community-Acquired Pneumonia Requiring Hospitalization among U.S. Children

(2015) Seema Jain et al.   NEW ENGLAND JOURNAL OF MEDICINE

Pompe disease: from pathophysiology to therapy and back again

(2014) Jeong-A Lim et al.   Frontiers in Aging Neuroscience

Pompe Disease: Early Diagnosis and Early Treatment Make a Difference

(2013) Yin-Hsiu Chien et al.   Pediatrics and Neonatology

The impact of antibodies in late-onset Pompe disease: A case series and literature review

(2012) Trusha T. Patel et al.   MOLECULAR GENETICS AND METABOLISM

The quick motor function test: a new tool to rate clinical severity and motor function in Pompe patients

(2011) Carine I. van Capelle et al.   JOURNAL OF INHERITED METABOLIC DISEASE

THE GROSS MOTOR FUNCTION MEASURE: A MEANS TO EVALUATE THE EFFECTS OF PHYSICAL THERAPY

(2010) Dianne J. Russell et al.   DEVELOPMENTAL MEDICINE AND CHILD NEUROLOGY

Recommendations for Quantification Methods During the Performance of a Pediatric Echocardiogram: A Report From the Pediatric Measurements Writing Group of the American Society of Echocardiography Pediatric and Congenital Heart Disease Council

(2010) Leo Lopez et al.   JOURNAL OF THE AMERICAN SOCIETY OF ECHOCARDIOGRAPHY

Effect of enzyme therapy in juvenile patients with Pompe disease: A three-year open-label study

(2010) C.I. van Capelle et al.   NEUROMUSCULAR DISORDERS

A Randomized Study of Alglucosidase Alfa in Late-Onset Pompe's Disease

(2010) Ans T. van der Ploeg et al.   NEW ENGLAND JOURNAL OF MEDICINE

Clinical outcomes after long-term treatment with alglucosidase alfa in infants and children with advanced Pompe disease

(2009) Marc Nicolino et al.   GENETICS IN MEDICINE

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

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

Management and treatment of glycogenosis type II

(2008) B. Bembi et al.   NEUROLOGY

Rate of disease progression during long-term follow-up of patients with late-onset Pompe disease

(2008) N.A.M.E. Van der Beek et al.   NEUROMUSCULAR DISORDERS

Reference Curves for the Gross Motor Function Measure: Percentiles for Clinical Description and Tracking Over Time Among Children With Cerebral Palsy

(2008) Steven E Hanna et al.   PHYSICAL THERAPY