welcome to DuchenneXchange
- a positively charged Duchenne muscular dystrophy community.- join today!
- login
A 48-Week, Open Label, Study to Evaluate the Efficacy and Safety of Casimersen, Eteplirsen and Golodirsen in Subjects With Duchenne Muscular Dystrophy Carrying Eligible DMD Duplications
study id #: NCT04179409
condition: Duchenne Muscular Dystrophy
status: not yet recruiting
purpose:This is an 48-week open-label study to determine the efficacy and safety of casimersen, eteplirsen, or golodirsen for the treatment of boys with duchenne muscular dystrophy who have a single exon duplication of either exon 45, 51 or 53, respectively. There will be weekly infusions and two muscle biopsies at baseline and at month 12.
intervention: Casimersen, Eteplirsen, Golodirsen
mechanism of action: Exon-skipping to promote dystrophin production
results: https://clinicaltrials.gov/ct2/show/results/NCT04179409
last updated: December 04, 2019
rareRelated
-
Wave Life Sciences Announces Suvodirsen Phase 1 Safety and Tolerability Data and Phase 2/3 Clinical Trial DesignWave Life Sciences Ltd., a clinical-stag...
-
A Study of the Safety, Tolerability & Efficacy of Long-term Administration of Drisapersen in US & Canadian S...This is a phase III, multicenter, open-l...
-
Pharmacokinetic Properties of Chronic Administration of Eteplirsen in the Treatment of Boys with Duchenne Muscular D...Objective: The objective of this analys...
-
FDA paves way for clinical trials of Sarepta’s Duchenne MD therapy SRP-5051The U.S. Food and Drug Administration ha...
-
Local boy with Duchenne muscular dystrophy begins promising gene-targeted therapySeven-year-old Wyatt Hubbard was a bit t...
-
Open Label Study of GSK2402968 in Subjects With Duchenne Muscular DystrophyThe purpose of this study is to explore ...
-
Restoring Dystrophin Expression in Duchenne Muscular Dystrophy: Current Status of Therapeutic ApproachesDuchenne muscular dystrophy (DMD), a rar...