Safety and efficacy of cipaglucosidase alfa plus miglustat versus alglucosidase alfa plus placebo in late-onset Pompe disease (PROPEL): an international, randomised, double-blind, parallel-group, phase 3 trial

The Lancet Neurology ◽

10.1016/s1474-4422(21)00331-8 ◽

2021 ◽

Vol 20 (12) ◽

pp. 1027-1037 ◽

Author(s):

Benedikt Schoser ◽

Mark Roberts ◽

Barry J Byrne ◽

Sheela Sitaraman ◽

Hai Jiang ◽

...

Keyword(s):

Pompe Disease ◽

Phase 3 ◽

Double Blind ◽

Safety And Efficacy ◽

Parallel Group ◽

Alglucosidase Alfa

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Safety and efficacy of avalglucosidase alfa versus alglucosidase alfa in patients with late-onset Pompe disease (COMET): a phase 3, randomised, multicentre trial

The Lancet Neurology ◽

10.1016/s1474-4422(21)00241-6 ◽

2021 ◽

Vol 20 (12) ◽

pp. 1012-1026 ◽

Author(s):

Jordi Diaz-Manera ◽

Priya S Kishnani ◽

Hani Kushlaf ◽

Shafeeq Ladha ◽

Tahseen Mozaffar ◽

...

Keyword(s):

Pompe Disease ◽

Multicentre Trial ◽

Phase 3 ◽

Safety And Efficacy ◽

Alglucosidase Alfa

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T.O.4 Safety and efficacy results from a randomized, double-blind, placebo-controlled study of alglucosidase alfa for the treatment of Pompe disease in juveniles and adults

Neuromuscular Disorders ◽

10.1016/j.nmd.2008.06.379 ◽

2008 ◽

Vol 18 (9-10) ◽

pp. 832-833 ◽

Author(s):

P. Laforet ◽

P.R. Clemens ◽

D. Corzo ◽

D. Escolar ◽

J. Florence ◽

...

Keyword(s):

Pompe Disease ◽

Controlled Study ◽

Double Blind ◽

Safety And Efficacy ◽

Double Blind Placebo ◽

Alglucosidase Alfa

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Safety and Efficacy of A-101 Hydrogen Peroxide Topical Solution in Adults with Seborrheic Keratosis: Results from the Phase 3, Randomized, Double-Blind, Vehicle-Controlled, Parallel-Group Study

SKIN The Journal of Cutaneous Medicine ◽

10.25251/skin.1.supp.118 ◽

2017 ◽

Vol 1 ◽

pp. s119 ◽

Author(s):

Zoe D Draelos ◽

Steven E Kempers ◽

Stacy R Smith ◽

David C Wilson ◽

Christopher V Powala ◽

...

Keyword(s):

Hydrogen Peroxide ◽

Seborrheic Keratosis ◽

Phase 3 ◽

Double Blind ◽

Safety And Efficacy ◽

Parallel Group Study ◽

Parallel Group ◽

Topical Solution

Abstract Not Available Disclosures: Study supported by Alcaris.

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Late-onset pompe disease: Case report of a patient with an uncommon mutation and observational clinical trial as to response to enzyme replacemant therapy (ERT) with alglucosidase alfa over a 12-month-period

Klinische Neurophysiologie ◽

10.1055/s-0030-1250913 ◽

2010 ◽

Vol 41 (01) ◽

Author(s):

N Strigl-Pill

Keyword(s):

Clinical Trial ◽

Case Report ◽

Pompe Disease ◽

Disease Case ◽

Alglucosidase Alfa

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Faculty Opinions recommendation of Efficacy and safety of sirukumab in patients with active rheumatoid arthritis refractory to anti-TNF therapy (SIRROUND-T): a randomised, double-blind, placebo-controlled, parallel-group, multinational, phase 3 study.

Faculty Opinions – Post-Publication Peer Review of the Biomedical Literature ◽

10.3410/f.727328077.793564275 ◽

2019 ◽

Author(s):

Ladislav Senolt

Keyword(s):

Rheumatoid Arthritis ◽

Active Rheumatoid Arthritis ◽

Efficacy And Safety ◽

Phase 3 ◽

Double Blind ◽

Double Blind Placebo ◽

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Spine Treatment Appraisal Report (STAR): Does Riluzole Improve Outcomes in Patients Undergoing Decompression Surgery for Degenerative Cervical Myelopathy?

Global Spine Journal ◽

10.1177/2192568221997401 ◽

2021 ◽

pp. 219256822199740

Author(s):

Joseph R. Dettori

Keyword(s):

Cervical Myelopathy ◽

Decompressive Surgery ◽

Decompression Surgery ◽

Phase 3 ◽

Double Blind ◽

Safety And Efficacy ◽

Double Blind Placebo ◽

Degenerative Cervical Myelopathy

Fehlings MG, Badhiwala JH, Ahn H, et al. Safety and efficacy of riluzole in patients undergoing decompressive surgery for degenerative cervical myelopathy (CSM-Protect): a multicentre, double-blind, placebo-controlled, randomised, phase 3 trial. Lancet Neurol. 2020.

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A phase 3, randomized, double-blind, parallel-group study to evaluate tezacaftor/ivacaftor in people with cystic fibrosis heterozygous for F508del-CFTR and a gating mutation

Journal of Cystic Fibrosis ◽

10.1016/j.jcf.2020.11.003 ◽

2020 ◽

Author(s):

Edward F. McKone ◽

Emily A. DiMango ◽

Sivagurunathan Sutharsan ◽

Tara Lynn Barto ◽

Daniel Campbell ◽

...

Keyword(s):

Cystic Fibrosis ◽

Phase 3 ◽

Double Blind ◽

Parallel Group Study ◽

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Efficacy and Safety of the Biosimilar IBI301 Plus Standard CHOP (I-CHOP) in Comparison With Rituximab Plus CHOP (R-CHOP) in Patients with Previously Untreated Diffuse Large B-Cell Lymphoma (DLBCL): A Randomized, Double-Blind, Parallel-Group, Phase 3 Trial

Advances in Therapy ◽

10.1007/s12325-020-01603-8 ◽

2021 ◽

Vol 38 (4) ◽

pp. 1889-1903

Author(s):

Yuqin Song ◽

Hui Zhou ◽

Huilai Zhang ◽

Wei Liu ◽

Yuerong Shuang ◽

...

Keyword(s):

B Cell ◽

Cell Lymphoma ◽

B Cell Lymphoma ◽

Efficacy And Safety ◽

Phase 3 ◽

Double Blind ◽

Large B Cell Lymphoma ◽

Parallel Group ◽

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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 ◽

Single Centre ◽

Enzyme Replacement ◽

Follow Up Study ◽

Alglucosidase Alfa

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Safety and efficacy of riluzole in patients undergoing decompressive surgery for degenerative cervical myelopathy (CSM-Protect): a multicentre, double-blind, placebo-controlled, randomised, phase 3 trial

The Lancet Neurology ◽

10.1016/s1474-4422(20)30407-5 ◽

2020 ◽

Author(s):

Michael G Fehlings ◽

Jetan H Badhiwala ◽

Henry Ahn ◽

H Francis Farhadi ◽

Christopher I Shaffrey ◽

...

Keyword(s):

Cervical Myelopathy ◽

Decompressive Surgery ◽

Phase 3 ◽

Double Blind ◽

Safety And Efficacy ◽

Double Blind Placebo ◽

Degenerative Cervical Myelopathy

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