P.11.21 Bioanalysis of a double blind, placebo-controlled clinical phase 2 study of drisapersen for the treatment of boys suffering from Duchenne muscular dystrophy and comparison to clinical outcome results

Muscular Dystrophy ◽

Clinical Outcome ◽

Phase 2 ◽

Double Blind ◽

Double Blind Placebo ◽

Clinical Phase ◽

Treatment of Duchenne muscular dystrophy with cyclosporin A – A randomized, double-blind, placebo controlled trial

Neuropediatrics ◽

10.1055/s-0029-1215757 ◽

2008 ◽

Vol 39 (05) ◽

Author(s):

J Kirschner ◽

J Schessl ◽

G Ihorst ◽

R Korinthenberg

Keyword(s):

Muscular Dystrophy ◽

Cyclosporin A ◽

Controlled Trial ◽

Double Blind ◽

Double Blind Placebo

Ataluren in Patients Aged  2 to < 5 Years with Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD): 28-week Results from a Phase 2 Study

10.1055/s-0039-1698256 ◽

2019 ◽

Author(s):

Christian Werner ◽

Cuixia Tian ◽

Robert Kong ◽

Edward O’Mara ◽

Traci Schilling ◽

...

Keyword(s):

Muscular Dystrophy ◽

Nonsense Mutation ◽

Phase 2 ◽

A Phase 2 Study for Dose Determination of SRP-5051, Then Dose Expansion in Patients With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment

Case Medical Research ◽

10.31525/ct1-nct04004065 ◽

2019 ◽

Author(s):

Keyword(s):

Muscular Dystrophy ◽

Phase 2 ◽

Dose Determination ◽

Faculty Opinions recommendation of P2X3 receptor antagonist (AF-219) in refractory chronic cough: a randomised, double-blind, placebo-controlled phase 2 study.

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

10.3410/f.725255169.793503497 ◽

2015 ◽

Author(s):

Brendan J Canning

Keyword(s):

Receptor Antagonist ◽

Chronic Cough ◽

Phase 2 ◽

P2x3 Receptor ◽

Double Blind ◽

Double Blind Placebo ◽

Faculty Opinions recommendation of Efficacy and safety of regorafenib in adult patients with metastatic osteosarcoma: a non-comparative, randomised, double-blind, placebo-controlled, phase 2 study.

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

10.3410/f.734506216.793579336 ◽

2020 ◽

Author(s):

Nathalie Gaspar

Keyword(s):

Adult Patients ◽

Phase 2 ◽

Efficacy And Safety ◽

Double Blind ◽

Double Blind Placebo ◽

Metastatic Osteosarcoma ◽

Anlotinib vs placebo as third- or further-line treatment for patients with small cell lung cancer: a randomised, double-blind, placebo-controlled Phase 2 study

British Journal of Cancer ◽

10.1038/s41416-021-01356-3 ◽

2021 ◽

Author(s):

Ying Cheng ◽

Qiming Wang ◽

Kai Li ◽

Jianhua Shi ◽

Ying Liu ◽

...

Keyword(s):

Lung Cancer ◽

Small Cell Lung Cancer ◽

Cell Lung Cancer ◽

Small Cell ◽

Line Treatment ◽

Phase 2 ◽

Small Cell Lung ◽

Double Blind ◽

Double Blind Placebo ◽

P.11.18 Design of a confirmatory phase 3, multicenter, randomized, double-blind, placebo-controlled study of ataluren in patients with nonsense mutation Duchenne muscular dystrophy

Neuromuscular Disorders ◽

10.1016/j.nmd.2013.06.579 ◽

2013 ◽

Vol 23 (9-10) ◽

pp. 804

Author(s):

J. Barth ◽

A. Reha ◽

R. Spiegel ◽

G.L. Elfring ◽

M. Husain ◽

...

Keyword(s):

Muscular Dystrophy ◽

Nonsense Mutation ◽

Controlled Study ◽

Phase 3 ◽

Double Blind ◽

Double Blind Placebo ◽

Confirmatory Phase

SAT0110 Efficacy and Safety of Pateclizumab (Anti-Lymphotoxin-Alpha) in Dmard-Ir Patients: Results of a Randomized, Double-Blind, Placebo-Controlled Head-To-Head Phase 2 Study with Adalimumab

Annals of the Rheumatic Diseases ◽

10.1136/annrheumdis-2013-eular.1836 ◽

2013 ◽

Vol 72 (Suppl 3) ◽

pp. A617.2-A618

Author(s):

W. Kennedy ◽

P. Horn ◽

Z. Su ◽

K. Basu ◽

A. Herman ◽

...

Keyword(s):

Phase 2 ◽

Efficacy And Safety ◽

Double Blind ◽

Double Blind Placebo ◽

Phase 2 Study ◽

Lymphotoxin Alpha

Faculty Opinions recommendation of Risankizumab, an IL-23 inhibitor, for ankylosing spondylitis: results of a randomised, double-blind, placebo-controlled, proof-of-concept, dose-finding phase 2 study.

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

10.3410/f.733519001.793576228 ◽

2020 ◽

Author(s):

Pedro Machado

Keyword(s):

Ankylosing Spondylitis ◽

Dose Finding ◽

Phase 2 ◽

Proof Of Concept ◽

Double Blind ◽

Double Blind Placebo ◽

P.337Dystrophin restoration by exon 53 skipping in patients with Duchenne muscular dystrophy after viltolarsen treatment: phase 2 study update

Neuromuscular Disorders ◽

10.1016/j.nmd.2019.06.451 ◽

2019 ◽

Vol 29 ◽

pp. S165-S166 ◽

Cited By ~ 1

Author(s):

P. Clemens ◽

V. Rao ◽

A. Connolly ◽

C. Zaidman ◽

A. Harper ◽

...

Keyword(s):

Muscular Dystrophy ◽

Treatment Phase ◽

Phase 2 ◽