peer reviewedThe aim of this Phase 1/2, 2-part, multicenter trial was to report clinical safety and efficacy of long-term golodirsen treatment among ambulatory patients with exon 53 skip-amenable Duchenne muscular dystrophy (DMD). Part 1 was a 12-week, randomized, double-blind, placebo-controlled, dose-titration study followed by 9-week safety review. Part 2 was a 168-week, open-label evaluation of golodirsen 30 mg/kg. Part 1 primary endpoint was safety. Part 2 primary endpoints were dystrophin protein expression and 6-minute walk test (6MWT); secondary endpoints were percent predicted forced vital capacity (FVC%p) and safety. Post hoc ambulation analyses used mutation-matched external natural history controls. All patients from Part 1 (gol...
<div><p>Background</p><p>Drisapersen induces exon 51 skipping during dystrophin pre-mRNA splicing an...
With the emergence of experimental therapies for Duchenne muscular dystrophy (DMD), it is fundamenta...
The aim of this international collaborative effort was to report 36-month longitudinal changes using...
The aim of this Phase 1/2, 2-part, multicenter trial was to report clinical safety and efficacy of l...
peer reviewedOBJECTIVE: To report safety, pharmacokinetics, exon 53 skipping, and dystrophin express...
Objective: To report safety, pharmacokinetics, exon 53 skipping, and dystrophin expression in golodi...
Objective To report safety, pharmacokinetics, exon 53 skipping, and dystrophin expression in golodir...
To continue evaluation of the long-term efficacy and safety of eteplirsen, a phosphorodiamidate morp...
BACKGROUND: Duchenne muscular dystrophy (DMD) is a rare, genetic disease caused by mutations in the ...
BACKGROUND: Duchenne muscular dystrophy (DMD) is caused by DMD gene mutations, resulting in absence ...
BackgroundEteplirsen received accelerated FDA approval for treatment of Duchenne muscular dystrophy ...
Novel emerging therapies for Duchenne muscular dystrophy (DMD), such as antisense oligomer (AO) medi...
This 48-week, randomized, placebo-controlled phase 3 study (DMD114044; NCT01254019) evaluated effica...
Duchenne muscular dystrophy is a rare genetic disorder with life-limiting pathology. Drisapersen ind...
BackgroundDrisapersen induces exon 51 skipping during dystrophin pre-mRNA splicing and allows synthe...
<div><p>Background</p><p>Drisapersen induces exon 51 skipping during dystrophin pre-mRNA splicing an...
With the emergence of experimental therapies for Duchenne muscular dystrophy (DMD), it is fundamenta...
The aim of this international collaborative effort was to report 36-month longitudinal changes using...
The aim of this Phase 1/2, 2-part, multicenter trial was to report clinical safety and efficacy of l...
peer reviewedOBJECTIVE: To report safety, pharmacokinetics, exon 53 skipping, and dystrophin express...
Objective: To report safety, pharmacokinetics, exon 53 skipping, and dystrophin expression in golodi...
Objective To report safety, pharmacokinetics, exon 53 skipping, and dystrophin expression in golodir...
To continue evaluation of the long-term efficacy and safety of eteplirsen, a phosphorodiamidate morp...
BACKGROUND: Duchenne muscular dystrophy (DMD) is a rare, genetic disease caused by mutations in the ...
BACKGROUND: Duchenne muscular dystrophy (DMD) is caused by DMD gene mutations, resulting in absence ...
BackgroundEteplirsen received accelerated FDA approval for treatment of Duchenne muscular dystrophy ...
Novel emerging therapies for Duchenne muscular dystrophy (DMD), such as antisense oligomer (AO) medi...
This 48-week, randomized, placebo-controlled phase 3 study (DMD114044; NCT01254019) evaluated effica...
Duchenne muscular dystrophy is a rare genetic disorder with life-limiting pathology. Drisapersen ind...
BackgroundDrisapersen induces exon 51 skipping during dystrophin pre-mRNA splicing and allows synthe...
<div><p>Background</p><p>Drisapersen induces exon 51 skipping during dystrophin pre-mRNA splicing an...
With the emergence of experimental therapies for Duchenne muscular dystrophy (DMD), it is fundamenta...
The aim of this international collaborative effort was to report 36-month longitudinal changes using...