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6 results for “golodirsen”
Data from: Increased Dystrophin Production With Golodirsen in Patients with Duchenne Muscular Dystrophy
<p>Objective To report safety, pharmacokinetics, exon 53 skipping, and dystrophin expression in golodirsen-treated patients with Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping. Methods Part 1 was a randomized, double-blind, placebo-controlled, 12-week dose titration of once-weekly golodirsen; Part 2 is an ongoing, open-label evaluation. Safety and pharmacokinetics were primary and secondary objectives of Part 1. Primary biological outcome measures of part 2 were blinded exon skipping and dystrophin protein production on muscle biopsies (baseline, Week 48) evaluated, respectively using reverse transcription PCR and western blot and immunohistochemistry. Results Twelve patients were randomized to receive golodirsen (n=8) or placebo (n=4) in Part 1. All from Part 1 plus 13 additional patients received 30 mg/kg golodirsen in Part 2. Safety findings were consistent with those previously observed in pediatric DMD patients. Most of the study drug was excreted within 4 hours following administration. A significant increase in exon 53 skipping was associated with ~16-fold increase over baseline in dystrophin protein expression at Week 48, with a mean percent normal dystrophin protein standard of 1.019% (range, 0.09%-4.30%). Sarcolemmal localization of dystrophin was demonstrated by significantly increased dystrophin positive fibers (Week 48, p<0.001); and a positive correlation (Spearman-r=0.663; p<0.001) between dystrophin protein change from baseline, measured by western blot and immunohistochemistry.</p>
Study of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD)
ClinicalTrials.gov study NCT02500381. IPD Sharing: UNDECIDED. Countries: 24. Publications: 2.
A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice
ClinicalTrials.gov study NCT06606340. IPD Sharing: Not stated. Countries: 1. Publications: 0.
Data from: Increased dystrophin production with golodirsen in patients with Duchenne muscular dystrophy
Open the record for dataset details and reuse information.
An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy
ClinicalTrials.gov study NCT03532542. IPD Sharing: Not stated. Countries: 13. Publications: 0.
An Open-Label Study of Golodirsen in Non-Ambulant Patients With Duchenne Muscular Dystrophy
ClinicalTrials.gov study NCT04708314. IPD Sharing: Not stated. Countries: 1. Publications: 0.
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