G.P.3.05 Phase 2 study of PTC124 for nonsense mutation suppression therapy of Duchenne muscular dystrophy (DMD)

Neuromuscular Disorders ◽

10.1016/j.nmd.2007.06.080 ◽

2007 ◽

Vol 17 (9-10) ◽

pp. 783 ◽

Author(s):

C. Bönnemann ◽

R. Finkel ◽

B. Wong ◽

K. Flanigan ◽

J. Sampson ◽

...

Keyword(s):

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Nonsense Mutation ◽

Phase 2 ◽

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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):

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Nonsense Mutation ◽

Phase 2 ◽

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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):

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Phase 2 ◽

Dose Determination ◽

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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 ◽

Author(s):

P. Clemens ◽

V. Rao ◽

A. Connolly ◽

C. Zaidman ◽

A. Harper ◽

...

Keyword(s):

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Treatment Phase ◽

Phase 2 ◽

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A phase 2 trial of the safety and pharmacokinetics of ataluren in patients aged ≥2 to <5 years with nonsense mutation Duchenne muscular dystrophy

Neuromuscular Disorders ◽

10.1016/s0960-8966(18)30325-0 ◽

2018 ◽

Vol 28 ◽

pp. S12-S13

Author(s):

R. Kong ◽

E. O’Mara ◽

X. Luo ◽

P. Trifillis ◽

C. Werner ◽

...

Keyword(s):

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Nonsense Mutation ◽

Phase 2 ◽

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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

Neuromuscular Disorders ◽

10.1016/j.nmd.2013.06.582 ◽

2013 ◽

Vol 23 (9-10) ◽

pp. 805 ◽

Author(s):

A. Lourbakos ◽

C. Beekman ◽

T. Holling ◽

J. Testerink ◽

D. Duinsbergen ◽

...

Keyword(s):

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Clinical Outcome ◽

Phase 2 ◽

Double Blind ◽

Double Blind Placebo ◽

Clinical Phase ◽

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Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, randomised, placebo-controlled phase 2 study

The Lancet Neurology ◽

10.1016/s1474-4422(14)70195-4 ◽

2014 ◽

Vol 13 (10) ◽

pp. 987-996 ◽

Author(s):

Thomas Voit ◽

Haluk Topaloglu ◽

Volker Straub ◽

Francesco Muntoni ◽

Nicolas Deconinck ◽

...

Keyword(s):

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Phase 2 ◽

Safety And Efficacy ◽

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A phase 2 trial of the safety and pharmacokinetics of ataluren in patients aged 2 to 5 years with nonsense mutation Duchenne muscular dystrophy

Neuromuscular Disorders ◽

10.1016/j.nmd.2017.06.446 ◽

2017 ◽

Vol 27 ◽

pp. S218

Author(s):

P. Riebling ◽

R. Kong ◽

E. O'Mara ◽

X. Luo ◽

P. Trifillis ◽

...

Keyword(s):

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Nonsense Mutation ◽

Phase 2 ◽

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Ataluren: Clinical Trial Results in Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)

Neuropediatrics ◽

10.1055/s-0036-1583648 ◽

2016 ◽

Vol 47 (S 01) ◽

Author(s):

U. Schara ◽

C. McDonald ◽

K. Bushby ◽

M. Tulinius ◽

R. Finkel ◽

...

Keyword(s):

Clinical Trial ◽

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Nonsense Mutation ◽

Clinical Trial Results

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Disease-modifying effects of edasalonexent, an NF-κB inhibitor, in young boys with Duchenne muscular dystrophy: Results of the MoveDMD phase 2 and open label extension trial

Neuromuscular Disorders ◽

10.1016/j.nmd.2021.02.001 ◽

2021 ◽

Author(s):

Richard S. Finkel ◽

Erika Finanger ◽

Krista Vandenborne ◽

H. Lee Sweeney ◽

Gihan Tennekoon ◽

...

Keyword(s):

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Phase 2 ◽

Open Label Extension ◽

Disease Modifying ◽

Extension Trial

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The nonsense mutation stop+4 model correlates with motor changes in Duchenne muscular dystrophy

Neuromuscular Disorders ◽

10.1016/j.nmd.2021.02.015 ◽

2021 ◽

Author(s):

Claudia Brogna ◽

Giorgia Coratti ◽

Rachele Rossi ◽

Marcella Neri ◽

Sonia Messina ◽

...

Keyword(s):

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Nonsense Mutation

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