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MechanismDMD exon 51 inhibitors [+2] |
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Inactive Indication- |
Drug Highest PhasePhase 1/2 |
First Approval Ctry. / Loc.- |
First Approval Date20 Jan 1800 |
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MechanismDMD exon 51 modulators [+1] |
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Inactive Indication- |
Drug Highest PhaseDiscovery |
First Approval Ctry. / Loc.- |
First Approval Date20 Jan 1800 |
A Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study Assessing Safety, Tolerability, Pharmacodynamics, Efficacy, and Pharmacokinetics of DYNE-251 Administered to Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
The primary purpose of this study is to evaluate the safety, tolerability, and dystrophin protein levels in muscle tissue following multiple intravenous (IV) doses of DYNE-251 in participants with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping.
The study consists of 3 periods: a multiple-ascending dose (MAD) / placebo-controlled period (24 weeks), an open-label period (24 weeks) and a long-term extension (LTE) period (192 weeks).
100 Clinical Results associated with TfR1 x DMD exon 51
100 Translational Medicine associated with TfR1 x DMD exon 51
0 Patents (Medical) associated with TfR1 x DMD exon 51