welcome to DuchenneXchange
- a positively charged Duchenne muscular dystrophy community.- join today!
- log in
available
The Expanded Access Use of Viltolarsen in Duchenne Muscular Dystrophy With Confirmed Exon 53 Amenable Mutation
study id #: NCT04337112
condition: Muscular Dystrophy, Duchenne; DMD
status: available
purpose:This is an open label expanded access program for boys, 3 to 12 years old, for the treatment of Duchenne muscular dystrophy (DMD) with confirmed mutation(s) in the dystrophin gene that is amenable to skipping of exon 53.
intervention: Viltolarsen
mechanism of action: Exon-skipping to promote dystrophin production
last updated: April 21, 2020
study details
study contacts
rareRelated
-
Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMDThe main objective of this study is to e...
-
Rimeporide: safety, tolerability and pharmacokinetic results from a phase Ib study in DMD boys as well as explorator...In Duchenne muscular dystrophy, impaired...
-
A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy (HOPE-2)HOPE-2 is a double-blind clinical trial ...
-
Duchenne Muscular Dystrophy Tissue Bank for Exon SkippingWe will utilize the Cooperative Internat...
-
Tamoxifen in Duchenne Muscular DystrophyA randomised, double blind, placebo cont...
-
A Study to Assess the Efficacy and Safety of MNK-1411 in Duchenne Muscular DystrophyThis is a multicenter, double blind, pla...
-
Single Escalating Dose Pilot Trial of Canakinumab (ILARIS®) in Duchenne Muscular DystrophyCanakinumab is an anti-interleukin 1 bet...
-
Capricor Receives Rare Pediatric Disease Designation from FDA for CAP-1002 for Patients With Duchenne Muscular Dystr...Capricor Therapeutics, Inc., a biotechno...
-
Cardiac and Skeletal Muscle Effects in the Randomized HOPE-Duchenne TrialObjective: To assess the feasibility, s...
-
FibroGen Receives Fast Track Designation From the U.S. FDA for Pamrevlumab for the Treatment of Idiopathic Pulmonary...FibroGen, Inc., a biopharmaceutical comp...
-
Capricor Therapeutics Expands Exosome Technology for DMDCapricor Therapeutics is expanding the d...
