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

Replacement Therapy ◽

Pompe Disease ◽

Enzyme Replacement ◽

A 4-Year Observational Clinical Study of 24 Adult Pompe Disease Patients under Alglucosidase Alfa Enzyme Replacement Therapy

Journal of Neuromuscular Diseases ◽

10.3233/jnd-159058 ◽

2015 ◽

Vol 2 (s1) ◽

pp. S69-S69 ◽

Cited By ~ 1

Author(s):

Karolina M. Stepien ◽

Jane Whitby ◽

Mark Roberts ◽

Reena Sharma

Keyword(s):

Clinical Study ◽

Replacement Therapy ◽

Pompe Disease ◽

Enzyme Replacement ◽

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

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 ◽

Enzyme replacement therapy with alglucosidase alfa in Pompe disease: Clinical experience with rate escalation

10.1016/j.ymgme.2017.12.435 ◽

2018 ◽

Vol 123 (2) ◽

pp. 92-96 ◽

Cited By ~ 3

Author(s):

Ankit K. Desai ◽

Crista K. Walters ◽

Heidi L. Cope ◽

Zoheb B. Kazi ◽

Stephanie M. DeArmey ◽

...

Keyword(s):

Replacement Therapy ◽

Clinical Experience ◽

Pompe Disease ◽

Enzyme Replacement ◽

Observational clinical study of 22 adult-onset Pompe disease patients undergoing enzyme replacement therapy over 5years

10.1016/j.ymgme.2016.01.013 ◽

2016 ◽

Vol 117 (4) ◽

pp. 413-418 ◽

Cited By ~ 24

Author(s):

Karolina M. Stepien ◽

Christian J. Hendriksz ◽

Mark Roberts ◽

Reena Sharma

Keyword(s):

Clinical Study ◽

Replacement Therapy ◽

Pompe Disease ◽

Adult Onset ◽

Enzyme Replacement

Long‐term benefit of enzyme replacement therapy with alglucosidase alfa in adults with Pompe disease: Prospective analysis from the French Pompe Registry

Journal of Inherited Metabolic Disease ◽

10.1002/jimd.12272 ◽

2020 ◽

Vol 43 (6) ◽

pp. 1219-1231 ◽

Cited By ~ 1

Author(s):

Claudio Semplicini ◽

Marie De Antonio ◽

Nadjib Taouagh ◽

Anthony Béhin ◽

Françoise Bouhour ◽

...

Keyword(s):

Replacement Therapy ◽

Pompe Disease ◽

Prospective Analysis ◽

Enzyme Replacement ◽

Alglucosidase Alfa ◽

Term Benefit

Phase 1 safety and pharmacokinetics of the novel enzyme replacement therapy neoGAA in treatment-naïve and alglucosidase alfa-treated late-onset Pompe disease patients

10.1016/j.ymgme.2015.12.476 ◽

2016 ◽

Vol 117 (2) ◽

pp. S117-S118

Author(s):

Shafeeq Ladha ◽

Pascal Laforet ◽

Eugen Mengel ◽

Alan Pestronk ◽

Jean Pouget ◽

...

Keyword(s):

Replacement Therapy ◽

Pompe Disease ◽

Late Onset ◽

Phase 1 ◽

The Novel ◽

Enzyme Replacement ◽

Alglucosidase Alfa ◽

Treatment Naïve

Unwarranted, long term, alglucosidase alfa enzyme replacement therapy in two non-Pompe disease patients

Clinical Neurology and Neurosurgery ◽

10.1016/j.clineuro.2020.106048 ◽

2020 ◽

Vol 196 ◽

pp. 106048

Author(s):

Chafic Karam ◽

Thomas Ragole ◽

Orly Moshe-Lilie ◽

Nizar Chahin

Keyword(s):

Replacement Therapy ◽

Pompe Disease ◽

Enzyme Replacement ◽

Phase 1 exploratory efficacy of the novel enzyme replacement therapy neoGAA in treatment-naïve and alglucosidase alfa-treated late-onset Pompe disease patients

10.1016/j.ymgme.2015.12.396 ◽

2016 ◽

Vol 117 (2) ◽

pp. S92

Author(s):

Loren Pena ◽

Richard Baron ◽

Barry Byrne ◽

Claude Desnuelle ◽

Ozlem Goker-Alpan ◽

...

Keyword(s):

Replacement Therapy ◽

Pompe Disease ◽

Late Onset ◽

Phase 1 ◽

The Novel ◽

Enzyme Replacement ◽

Alglucosidase Alfa ◽

Treatment Naïve

Cost-effectiveness of enzyme replacement therapy with alglucosidase alfa in classic-infantile patients with Pompe disease

Orphanet Journal of Rare Diseases ◽

10.1186/1750-1172-9-75 ◽

2014 ◽

Vol 9 (1) ◽

pp. 75 ◽

Cited By ~ 20

Author(s):

Tim A Kanters ◽

Iris Hoogenboom-Plug ◽

Maureen PMH Rutten-Van Mölken ◽

W Redekop ◽

Ans T van der Ploeg ◽

...

Keyword(s):

Cost Effectiveness ◽

Replacement Therapy ◽

Pompe Disease ◽

Enzyme Replacement ◽

Strategy to assess the effect of duvoglustat co-administered with alglucosidase alfa infusion on the immune response to enzyme replacement therapy for Pompe disease

10.1016/j.ymgme.2013.12.038 ◽

2014 ◽

Vol 111 (2) ◽

pp. S25

Author(s):

Elfrida R. Benjamin ◽

Xiaoyang Wu ◽

M. Cecilia Della Valle ◽

Eric Sjoberg ◽

Sheela Sitaraman ◽

...

Keyword(s):

Immune Response ◽

Replacement Therapy ◽

Pompe Disease ◽

Enzyme Replacement ◽