Quality of life and participation in daily life of adults with Pompe disease receiving enzyme replacement therapy: 10 years of international follow-up

How to describe the clinical spectrum in Pompe disease?

(2013) Deniz Güngör et al.   AMERICAN JOURNAL OF MEDICAL GENETICS PART A

Enzyme replacement therapy and fatigue in adults with Pompe disease

(2013) Deniz Güngör et al.   MOLECULAR GENETICS AND METABOLISM

Pain in adult patients with Pompe disease

(2013) D. Güngör et al.   MOLECULAR GENETICS AND METABOLISM

Impact of enzyme replacement therapy on survival in adults with Pompe disease: results from a prospective international observational study

(2013) Deniz Güngör et al.   Orphanet Journal of Rare Diseases

36 months observational clinical study of 38 adult Pompe disease patients under alglucosidase alfa enzyme replacement therapy

(2012) Caroline Regnery et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Enzyme replacement therapy in late-onset Pompe disease: a systematic literature review

(2012) Antonio Toscano et al.   JOURNAL OF NEUROLOGY

Open-label extension study following the Late-Onset Treatment Study (LOTS) of alglucosidase alfa

(2012) Ans T. van der Ploeg et al.   MOLECULAR GENETICS AND METABOLISM

The Rasch-built Pompe-specific Activity (R-PAct) scale

(2012) N.A.M.E. van der Beek et al.   NEUROMUSCULAR DISORDERS

Effect of enzyme therapy and prognostic factors in 69 adults with Pompe disease: an open-label single-center study

(2012) Juna M de Vries et al.   Orphanet Journal of Rare Diseases

24-Months results in two adults with Pompe disease on enzyme replacement therapy

(2011) Stefan Vielhaber et al.   CLINICAL NEUROLOGY AND NEUROSURGERY

Burden of illness of Pompe disease in patients only receiving supportive care

(2011) Tim A. Kanters et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Observational clinical study in juvenile-adult glycogenosis type 2 patients undergoing enzyme replacement therapy for up to 4 years

(2011) C. Angelini et al.   JOURNAL OF NEUROLOGY

Recombinant human acid alpha-glucosidase (rhGAA) in adult patients with severe respiratory failure due to Pompe disease

(2011) David Orlikowski et al.   NEUROMUSCULAR DISORDERS

Long-term observational, non-randomized study of enzyme replacement therapy in late-onset glycogenosis type II

(2010) Bruno Bembi et al.   JOURNAL OF INHERITED METABOLIC DISEASE

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

Enzyme replacement therapy with alglucosidase alfa in 44 patients with late-onset glycogen storage disease type 2: 12-month results of an observational clinical trial

(2009) S. Strothotte et al.   JOURNAL OF NEUROLOGY

Progress in enzyme replacement therapy in glycogen storage disease type II

(2009) Corrado Angelini et al.   Therapeutic Advances in Neurological Disorders

Glycogen storage disease type II (Pompe disease) - influence of enzyme replacement therapy in adults

(2008) T. Merk et al.   EUROPEAN JOURNAL OF NEUROLOGY

Clinical features of late-onset Pompe disease: A prospective cohort study

(2008) John H.J. Wokke et al.   MUSCLE & NERVE