Long-term effects of enzyme replacement therapy in an elderly cohort of late-onset Pompe disease

Replacement Therapy ◽

Pompe Disease ◽

Late Onset ◽

Long Term Effects ◽

Enzyme Replacement ◽

Elderly Cohort

Muscle imaging data in late-onset Pompe disease reveal a correlation between the pre-existing degree of lipomatous muscle alterations and the efficacy of long-term enzyme replacement therapy

Molecular Genetics and Metabolism Reports ◽

10.1016/j.ymgmr.2015.03.010 ◽

2015 ◽

Vol 3 ◽

pp. 58-64 ◽

Cited By ~ 13

Author(s):

Kai Michael Gruhn ◽

Christoph Malte Heyer ◽

Anne-Katrin Güttsches ◽

Robert Rehmann ◽

Volkmar Nicolas ◽

...

Keyword(s):

Replacement Therapy ◽

Pompe Disease ◽

Late Onset ◽

Imaging Data ◽

Respiratory function in late onset Pompe disease patients upon long-term enzyme replacement therapy

Klinische Neurophysiologie ◽

10.1055/s-0032-1301655 ◽

2012 ◽

Vol 43 (01) ◽

Cited By ~ 1

Author(s):

I Schneider ◽

F Hanisch ◽

T Müller ◽

S Zierz

Keyword(s):

Replacement Therapy ◽

Pompe Disease ◽

Respiratory Function ◽

Late Onset ◽

Effect of long term enzyme replacement therapy in late onset Pompe disease: a single-centre experience

Neuromuscular Disorders ◽

10.1016/j.nmd.2020.12.001 ◽

2020 ◽

Author(s):

George K. Papadimas ◽

Christoforos Anagnostopoulos ◽

Sophia Xirou ◽

Helen Michelakakis ◽

Gerasimos Terzis ◽

...

Keyword(s):

Replacement Therapy ◽

Pompe Disease ◽

Late Onset ◽

Single Centre ◽

Enzyme Replacement ◽

Single Centre Experience

Respiratory function in late-onset Pompe disease patients receiving long-term enzyme replacement therapy for more than 48 months

Wiener Medizinische Wochenschrift ◽

10.1007/s10354-012-0153-5 ◽

2012 ◽

Vol 163 (1-2) ◽

pp. 40-44 ◽

Cited By ~ 18

Author(s):

Ilka Schneider ◽

Frank Hanisch ◽

Tobias Müller ◽

Bernd Schmidt ◽

Stephan Zierz

Keyword(s):

Replacement Therapy ◽

Pompe Disease ◽

Respiratory Function ◽

Late Onset ◽

Effects of short-to-long term enzyme replacement therapy (ERT) on skeletal muscle tissue in late onset Pompe disease (LOPD)

Neuropathology and Applied Neurobiology ◽

10.1111/nan.12414 ◽

2017 ◽

Vol 44 (5) ◽

pp. 449-462 ◽

Cited By ~ 11

Author(s):

M. Ripolone ◽

R. Violano ◽

D. Ronchi ◽

S. Mondello ◽

A. Nascimbeni ◽

...

Keyword(s):

Skeletal Muscle ◽

Replacement Therapy ◽

Muscle Tissue ◽

Pompe Disease ◽

Late Onset ◽

Skeletal Muscle Tissue ◽

Long-term enzyme-replacement therapy (ERT) with alglucosidase alfa: Evolution of two siblings with juvenile late-onset Pompe disease

Journal of the Neurological Sciences ◽

10.1016/j.jns.2015.08.007 ◽

2015 ◽

Vol 358 (1-2) ◽

pp. 459-460 ◽

Cited By ~ 1

Author(s):

J. Rafael Bretón Martínez ◽

Andrés Cánovas Martínez

Keyword(s):

Replacement Therapy ◽

Pompe Disease ◽

Late Onset ◽

Enzyme Replacement ◽

Alglucosidase Alfa

A 4-year follow-up study of 24 patients with late onset Pompe disease treated with alglucosidase alfa enzyme replacement therapy at a single centre

Molecular Genetics and Metabolism ◽

10.1016/j.ymgme.2014.12.252 ◽

2015 ◽

Vol 114 (2) ◽

pp. S111

Author(s):

Karolina M. Stepien ◽

Jane Whitby ◽

Mark Roberts ◽

Reena Sharma

Keyword(s):

Replacement Therapy ◽

Pompe Disease ◽

Late Onset ◽

Single Centre ◽

Enzyme Replacement ◽

Follow Up Study ◽

Alglucosidase Alfa

Long-Term Effects of Enzyme Replacement Therapy for Anderson-Fabry Disease

International Heart Journal ◽

10.1536/ihj.17-688 ◽

2019 ◽

Vol 60 (1) ◽

pp. 208-214

Author(s):

Miki Tsujiuchi ◽

Mio Ebato ◽

Hideyuki Maezawa ◽

Takuya Mizukami ◽

Ayaka Nogi ◽

...

Keyword(s):

Fabry Disease ◽

Replacement Therapy ◽

Long Term Effects ◽

Reduced Efficacy of Enzyme Replacement Therapy in a Child with Late-Onset Pompe Disease

Pediatrics & Therapeutics ◽

10.4172/2161-0665.1000290 ◽

2016 ◽

Vol 06 (02) ◽

Cited By ~ 1

Author(s):

Satoru Takahashi ◽

Ryosuke Tanaka

Keyword(s):

Replacement Therapy ◽

Pompe Disease ◽

Late Onset ◽

Long-term evaluation of respiratory outcomes in patients with late onset Pompe disease under enzyme replacement therapy: the role of postural drop and its impact on quality of life

10.1183/13993003.congress-2021.pa3291 ◽

2021 ◽

Author(s):

Grazia Crescimanno ◽

Luca Palazzolo ◽

Manuela Bertini ◽

Salvatore Arrisicato ◽

Oreste Marrone

Keyword(s):

Quality Of Life ◽

Pompe Disease ◽

Late Onset ◽

Enzyme Replacement ◽

Term Evaluation ◽

Respiratory Outcomes