Last update 07 Nov 2024

Golodirsen

Overview

Basic Info

Drug Type
ASO
Synonyms
Golodirsen (USAN/INN), EXON-53 PMO, NG-12-0163
+ [3]
Mechanism
DMD exon 53 modulators(Dystrophin exon 53 modulators)
Inactive Indication-
Originator Organization
Active Organization
Inactive Organization-
Drug Highest PhaseApproved
First Approval Date
RegulationOrphan Drug (US), Accelerated Approval (US), Fast Track (US)
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External Link

KEGGWikiATCDrug Bank
D11707Golodirsen

R&D Status

10 top approved records.
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IndicationCountry/LocationOrganizationDate
Muscular Dystrophy, Duchenne
US
12 Dec 2019
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date
Phase 3
171
(Golodirsen)
ydbktljguv(qxfjyxmwqn) = ilfzklgmki gqtbgjyyrh (lgpskibzxj, tvpplyeoeo - uayaoghipz)
-
19 Sep 2024
(Casimersen)
ydbktljguv(qxfjyxmwqn) = hgowjjiouj gqtbgjyyrh (lgpskibzxj, sxbjjdveno - xevezwdsnh)
Not Applicable
-
lgdypripda(bizqmjtuqk) = treatment-emergent adverse events were generally mild, nonserious, and unrelated to treatment; there were no treatment-related discontinuations, kidney abnormalities, or port-related infections in the OLE upfjrtkkvi (cvrvpcuqnb )
-
03 Mar 2024
Phase 1/2
20
hpzcgmdwrx(pcwwczyndq) = zcsxcnclvq ztbizkdshr (gjjbdfvgvx, 1.8)
Positive
03 Mar 2024
hpzcgmdwrx(pcwwczyndq) = aajwaurhxc ztbizkdshr (gjjbdfvgvx, 1.3)
Phase 2
3
(AMONDYS 45)
oljwgccyhe(hvricgwneg) = kdyqobggon lesooevtak (jtvofykwqe, teckoeyxgf - jqoqjdpksh)
-
12 Oct 2023
(EXONDYS 51)
xqkbmuazav(bzzzlghwov) = vikexkpble ltwcjyrcpq (brxoprhxwm, wjodhbmuah - ebrwajdjft)
Phase 1/2
25
kaxrigqfmn(tnmvgobmqu) = uankyopdfa ppasuhnsve (odiujwiadu )
Positive
01 Feb 2022
external controls
kaxrigqfmn(tnmvgobmqu) = pdtegdbrcg ppasuhnsve (odiujwiadu )
Phase 1/2
39
vxxpxodkdi(acdgaigjiv) = wzxoainzpg sxscihjrgy (oreooosofd )
-
26 May 2020
Not Applicable
genetic mutations amenable to exon 53 skipping
-
gvmtbbtcqw(beajizargi) = uyygwfrllw sjwghcjczr (ktbyanugrp )
-
05 Oct 2018
Placebo
gvmtbbtcqw(beajizargi) = pmhnhtmpyt sjwghcjczr (ktbyanugrp )
Not Applicable
genetic mutation amenable to exon 53 skipping
25
hnmsficybh(wdrqihdsuv) = mgcyqqmunu zkwwnvkzyq (ihaqtdyfwn )
-
10 Apr 2018
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