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

Loren Pena; Richard Baron; Barry Byrne; Claude Desnuelle; Ozlem Goker-Alpan; Shafeeq Ladha; Pascal Laforet; Eugen Mengel; Alan Pestronki; Jean Pouget; Benedikt Schoser; Volker Straub; Jaya Trivedi; Philip Van Damme; John Vissing; Peter Young; Beth Thurberg; Kerry Culm-Merdek; Gerard Short; Ans van der Ploeg

doi:10.1016/j.ymgme.2015.12.396

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

Molecular Genetics and Metabolism ◽

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):

Enzyme Replacement Therapy ◽

Replacement Therapy ◽

Pompe Disease ◽

Late Onset ◽

Phase 1 ◽

The Novel ◽

Enzyme Replacement ◽

Alglucosidase Alfa ◽

Treatment Naïve

Download Full-text

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

Molecular Genetics and Metabolism ◽

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):

Enzyme Replacement Therapy ◽

Replacement Therapy ◽

Pompe Disease ◽

Late Onset ◽

Phase 1 ◽

The Novel ◽

Enzyme Replacement ◽

Alglucosidase Alfa ◽

Treatment Naïve

Download Full-text

Safety, tolerability, pharmacokinetics, pharmacodynamics, and exploratory efficacy of the novel enzyme replacement therapy avalglucosidase alfa (neoGAA) in treatment-naïve and alglucosidase alfa-treated patients with late-onset Pompe disease: A phase 1, open-label, multicenter, multinational, ascending dose study

Neuromuscular Disorders ◽

10.1016/j.nmd.2018.12.004 ◽

2019 ◽

Vol 29 (3) ◽

pp. 167-186 ◽

Cited By ~ 9

Author(s):

Loren D.M. Pena ◽

Richard J. Barohn ◽

Barry J. Byrne ◽

Claude Desnuelle ◽

Ozlem Goker-Alpan ◽

...

Keyword(s):

Enzyme Replacement Therapy ◽

Pompe Disease ◽

Late Onset ◽

Phase 1 ◽

The Novel ◽

Open Label ◽

Enzyme Replacement ◽

Alglucosidase Alfa ◽

Dose Study ◽

Treatment Naïve

Download Full-text

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):

Enzyme Replacement Therapy ◽

Replacement Therapy ◽

Pompe Disease ◽

Late Onset ◽

Single Centre ◽

Enzyme Replacement ◽

Follow Up Study ◽

Alglucosidase Alfa

Download Full-text

Effect of enzyme replacement therapy with alglucosidase alfa (Myozyme®) in 12 patients with advanced late-onset Pompe disease

Molecular Genetics and Metabolism ◽

10.1016/j.ymgme.2017.06.007 ◽

2017 ◽

Vol 122 (1-2) ◽

pp. 80-85 ◽

Cited By ~ 14

Author(s):

Constantinos Papadopoulos ◽

David Orlikowski ◽

Hélène Prigent ◽

Arnaud Lacour ◽

Céline Tard ◽

...

Keyword(s):

Enzyme Replacement Therapy ◽

Replacement Therapy ◽

Pompe Disease ◽

Late Onset ◽

Enzyme Replacement ◽

Alglucosidase Alfa

Download Full-text

Effect of enzyme replacement therapy with alglucosidase alfa (Myozyme®) in 12 patients with advanced late-onset Pompe disease

Neuromuscular Disorders ◽

10.1016/j.nmd.2016.06.089 ◽

2016 ◽

Vol 26 ◽

pp. S109-S110 ◽

Cited By ~ 2

Author(s):

C. Papadopoulos ◽

D. Orlikowski ◽

H. Prigent ◽

B. Perniconi ◽

N. Taouagh ◽

...

Keyword(s):

Enzyme Replacement Therapy ◽

Replacement Therapy ◽

Pompe Disease ◽

Late Onset ◽

Enzyme Replacement ◽

Alglucosidase Alfa

Download Full-text

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):

Enzyme Replacement Therapy ◽

Replacement Therapy ◽

Pompe Disease ◽

Late Onset ◽

Enzyme Replacement ◽

Alglucosidase Alfa

Download Full-text

Enzyme replacement therapy with alglucosidase alfa in a late-onset Pompe disease patient during pregnancy

Neuromuscular Disorders ◽

10.1016/j.nmd.2018.08.002 ◽

2018 ◽

Vol 28 (11) ◽

pp. 965-968 ◽

Cited By ~ 2

Author(s):

Miguel Oliveira Santos ◽

Teresinha Evangelista ◽

Isabel Conceição

Keyword(s):

Enzyme Replacement Therapy ◽

Replacement Therapy ◽

Pompe Disease ◽

Late Onset ◽

Disease Patient ◽

Enzyme Replacement ◽

Alglucosidase Alfa

Download Full-text

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):

Enzyme Replacement Therapy ◽

Replacement Therapy ◽

Pompe Disease ◽

Late Onset ◽

Enzyme Replacement

Download Full-text

FIVE-YEARS FOLLOW-UP OF THE RESPIRATORY AND MOTOR INVOLVEMENTS IN PATIENTS WITH LATE ONSET POMPE DISEASE (LOPD) ON IRREGULAR ENZYME REPLACEMENT THERAPY (ERT)

CHEST Journal ◽

10.1016/j.chest.2020.05.415 ◽

2020 ◽

Vol 157 (6) ◽

pp. A371

Author(s):

N. Sousa

Keyword(s):

Enzyme Replacement Therapy ◽

Replacement Therapy ◽

Pompe Disease ◽

Late Onset ◽

Enzyme Replacement

Download Full-text

Effects of enzyme replacement therapy on bone density in late onset Pompe disease

Molecular Genetics and Metabolism ◽

10.1016/j.ymgme.2019.11.034 ◽

2020 ◽

Vol 129 (2) ◽

pp. S24-S25

Author(s):

Mahima Avanti ◽

Virginia Kimonis ◽

Alice Chen

Keyword(s):

Bone Density ◽

Enzyme Replacement Therapy ◽

Replacement Therapy ◽

Pompe Disease ◽

Late Onset ◽

Enzyme Replacement

Download Full-text