선택지 정리됨

Duchenne muscular dystrophy (DMD): 국가별 선택지

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선택지 정리됨희귀·유전 질환최종 확인 2026.06

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임상시험 검색어

Duchenne muscular dystrophy (DMD) clinical trial

국가별 선택지

국가별 치료 선택지

공식 규제·평가 기관 출처를 바탕으로 한 국가별 승인·접근 상태입니다. 무엇이 어디에 존재하는지를 보여줄 뿐, 추천이 아닙니다.

United States

Givinostat / Duvyzat; vamorolone / Agamree; delandistrogene moxeparvovec-rokl / Elevidys; exon-skipping therapies including eteplirsen, golodirsen, viltolarsen, casimersen [1]FDA-approvedConfirmed DMD gene mutation for Elevidys; exon-specific amenability for exon-skipping drugs; all genetic variants for givinostat per FDA notice; no mutation-specific restriction captured for vamorolone; DMD patients aged 6 years and older for givinostat; DMD patients aged 2 years and older for vamorolone; ambulatory DMD patients aged 4 years and older with confirmed DMD mutation for current Elevidys-limited indication; confirmed exon-skipping amenability for antisense oligonucleotide therapies. · Elevidys has a boxed warning for serious liver injury/acute liver failure including fatal outcomes, and FDA removed the non-ambulatory indication in 2025. Exon-skipping drugs are mutation-specific and include accelerated-approval/confirmatory-study caveats in FDA review materials. Confidence/conflicts: High for U.S. regulatory status. Elevidys chronology is recorded as an updated regulatory restriction: 2025 revised label supersedes the broader 2024 expansion.
Givinostat / Duvyzat; vamorolone / Agamree; delandistrogene moxeparvovec-rokl / Elevidys; exon-skipping therapies including eteplirsen, golodirsen, viltolarsen, casimersen [1]FDA-approvedConfirmed DMD gene mutation for Elevidys; exon-specific amenability for exon-skipping drugs; all genetic variants for givinostat per FDA notice; no mutation-specific restriction captured for vamorolone; DMD patients aged 6 years and older for givinostat; DMD patients aged 2 years and older for vamorolone; ambulatory DMD patients aged 4 years and older with confirmed DMD mutation for current Elevidys-limited indication; confirmed exon-skipping amenability for antisense oligonucleotide therapies. · Elevidys has a boxed warning for serious liver injury/acute liver failure including fatal outcomes, and FDA removed the non-ambulatory indication in 2025. Exon-skipping drugs are mutation-specific and include accelerated-approval/confirmatory-study caveats in FDA review materials. Confidence/conflicts: High for U.S. regulatory status. Elevidys chronology is recorded as an updated regulatory restriction: 2025 revised label supersedes the broader 2024 expansion.
Givinostat / Duvyzat; vamorolone / Agamree; delandistrogene moxeparvovec-rokl / Elevidys; exon-skipping therapies including eteplirsen, golodirsen, viltolarsen, casimersen [1]FDA-approvedConfirmed DMD gene mutation for Elevidys; exon-specific amenability for exon-skipping drugs; all genetic variants for givinostat per FDA notice; no mutation-specific restriction captured for vamorolone; DMD patients aged 6 years and older for givinostat; DMD patients aged 2 years and older for vamorolone; ambulatory DMD patients aged 4 years and older with confirmed DMD mutation for current Elevidys-limited indication; confirmed exon-skipping amenability for antisense oligonucleotide therapies. · Elevidys has a boxed warning for serious liver injury/acute liver failure including fatal outcomes, and FDA removed the non-ambulatory indication in 2025. Exon-skipping drugs are mutation-specific and include accelerated-approval/confirmatory-study caveats in FDA review materials. Confidence/conflicts: High for U.S. regulatory status. Elevidys chronology is recorded as an updated regulatory restriction: 2025 revised label supersedes the broader 2024 expansion.

European Union

Vamorolone / Agamree; givinostat / Duvyzat [2]EMA authorisedNo mutation-specific restriction for Agamree/vamorolone; Duvyzat/givinostat indicated for ambulant patients aged 6 years and older with concomitant corticosteroid treatment; DMD from age 4 for vamorolone; ambulant DMD aged 6 years and older with corticosteroids for givinostat/Duvyzat. · EMA Duvyzat authorisation is conditional and requested further studies. NICE guidance is national reimbursement/appraisal context and should not be generalized to all EU countries. Confidence/conflicts: High for EMA/NICE status. No source conflict identified.
Vamorolone / Agamree; givinostat / Duvyzat [2]EMA authorisedNo mutation-specific restriction for Agamree/vamorolone; Duvyzat/givinostat indicated for ambulant patients aged 6 years and older with concomitant corticosteroid treatment; DMD from age 4 for vamorolone; ambulant DMD aged 6 years and older with corticosteroids for givinostat/Duvyzat. · EMA Duvyzat authorisation is conditional and requested further studies. NICE guidance is national reimbursement/appraisal context and should not be generalized to all EU countries. Confidence/conflicts: High for EMA/NICE status. No source conflict identified.

Australia

corticosteroids; physiotherapy; scoliosis monitoring; cardiac and respiratory monitoring; mobility/assistive supports; palliative-care supports [3]Standard option (per Rare Awareness Rare Education Portal / Rare Voices Australia)pathogenic variants in the DMD gene / dystrophinopathy context; Australian DMD supportive and multidisciplinary care context across the disease course. · The RARE Portal is educational and states that it does not replace professional advice. It does not establish approval or subsidy for a branded DMD drug or gene therapy. Confidence/conflicts: Medium-high for Australian supportive-care categories; drug-specific regulator status remains separately source-pending.
corticosteroids; physiotherapy; scoliosis monitoring; cardiac and respiratory monitoring; mobility/assistive supports; palliative-care supports [3]Standard option (per Rare Awareness Rare Education Portal / Rare Voices Australia)pathogenic variants in the DMD gene / dystrophinopathy context; Australian DMD supportive and multidisciplinary care context across the disease course. · The RARE Portal is educational and states that it does not replace professional advice. It does not establish approval or subsidy for a branded DMD drug or gene therapy. Confidence/conflicts: Medium-high for Australian supportive-care categories; drug-specific regulator status remains separately source-pending.

Canada

vamorolone (Agamree)[4]Health Canada approvedDMD diagnosis; no mutation-specific restriction stated in the fetched Canadian Agamree indication; Canadian DMD treatment for patients aged 4 years and older. · Health Canada notes pediatric evidence is established for ages 4 to under 18, no geriatric indication is authorized, and the product has corticosteroid-related contraindications and monitoring considerations. This entry does not establish payer coverage. Confidence/conflicts: High for Canadian Agamree approval and age restriction; reimbursement implementation remains source-pending.
vamorolone (Agamree)[4]Health Canada approvedDMD diagnosis; no mutation-specific restriction stated in the fetched Canadian Agamree indication; Canadian DMD treatment for patients aged 4 years and older. · Health Canada notes pediatric evidence is established for ages 4 to under 18, no geriatric indication is authorized, and the product has corticosteroid-related contraindications and monitoring considerations. This entry does not establish payer coverage. Confidence/conflicts: High for Canadian Agamree approval and age restriction; reimbursement implementation remains source-pending.

출처

U.S. Food and Drug Administration — regulator safety and revised-label notice · regulator safety and revised-label notice
European Medicines Agency (EMA) — regulator EPAR · regulator EPAR
Rare Awareness Rare Education (RARE) Portal / Rare Voices Australia — Australian rare-disease educational portal · Australian rare-disease educational portal
Health Canada Drug and Health Product Portal — Summary Basis of Decision · Summary Basis of Decision

위 내용은 공식 규제·접근 상태일 뿐, 의학적 조언이나 추천이 아니고, 적격성을 판단하지도 않습니다. 어떤 선택지가 적합한지는 환자의 상황과 종양내과 팀에 달려 있습니다. 규제 상태는 바뀔 수 있으니 표시된 출처에서 확인하세요. 임상 세부 내용은 영문이 정본입니다. 최종 확인 2026-06-12.