뒤셴 근이영양증(DMD): 국가별 치료 선택지

임상시험 진행 중

• Vamorolone; prednisone comparator; idebenone; placebo임상시험 · NCT03439670임상시험Trial only (registry)United Kingdom · Genetically confirmed DMD for vamorolone study; no mutation restriction captured for idebenone respiratory-function study beyond DMD diagnosis; Ambulatory boys aged 4 to under 7 years with DMD for vamorolone study; DMD patients receiving glucocorticoid steroids with respiratory function decline criteria for idebenone study. · Idebenone study was terminated and should not be presented as available therapy. Vamorolone has separate EMA/NICE approval/access sources; this registry record is trial evidence only. Confidence/conflicts: High for registry statuses; idebenone is explicitly not recorded as current access. ClinicalTrials.gov — clinical-trial registry
• DYNE-251; casimersen / SRP-4045; golodirsen / SRP-4053; viltolarsen; delandistrogene moxeparvovec / SRP-9001; PF-06939926임상시험 · NCT05524883임상시험Trial only (registry)Korea · Exon 51 skipping for DYNE-251; exon 45 or exon 53 skipping for casimersen/golodirsen study; exon 53 skipping for viltolarsen studies; confirmed DMD mutation and AAV antibody criteria for gene-transfer studies; DMD amenable to specific exon skipping or gene-transfer trial criteria; many protocols require stable glucocorticoids, ambulatory status, or AAV antibody criteria. · Trial statuses include active-not-recruiting and completed. Genetic eligibility is protocol-specific; registry location does not imply marketed access in that country. Confidence/conflicts: High for registry cells; no approval claim is made for non-U.S. jurisdictions. ClinicalTrials.gov — clinical-trial registry
• SAT-3247임상시험임상시험Trial only (registry)Australia · confirmed DMD gene mutation required by trial eligibility; ambulatory male DMD patients aged 7 to under 10 years with documented clinical findings and prior genetic testing confirming a DMD mutation; study allows stable glucocorticoid/supportive-care contexts and has specific exclusions. · Trial registration does not establish approval, efficacy, access outside the study, or individual eligibility. Participation depends on site status, inclusion/exclusion criteria, consent, and investigator assessment. Confidence/conflicts: High for Australian registry-trial context; no approval or benefit claim is inferred. Australian New Zealand Clinical Trials Registry (ANZCTR) — clinical-trial registry
• ataluren (Translarna)임상시험임상시험InvestigationalAustralia · nonsense-mutation DMD context implied by ataluren mechanism, but TGA notice itself states DMD treatment indication broadly; Australian orphan-designation context for DMD; no current Australian approval was verified from this source. · Orphan designation is not the same as marketing approval, clinical availability, or payer coverage. The designation listed in the fetched notice has lapsed. Confidence/conflicts: High for lapsed orphan-designation status; no Australian approval was established. Therapeutic Goods Administration (TGA) — orphan-drug designation notice
• Ataluren / Translarna; NS-089/NCNP-02; DS-5141b임상시험 · NCT03179631임상시험Trial only (registry)Thailand · Nonsense point mutation in dystrophin gene for ataluren; Japan NS-089/NCNP-02 and DS-5141b extension studies require completion of prior molecule-specific studies; Nonsense-mutation DMD for ataluren; extension-study contexts for participants completing prior NS-089/NCNP-02 or DS-5141b studies. · Ataluren registry record is completed. Extension studies are limited to prior-study participants. Current authorization/reimbursement differs by country and remains source-pending outside verified sources. Confidence/conflicts: High for registry-listed trial cells; no approval claim is made. ClinicalTrials.gov — clinical-trial registry