Options mapped

Duchenne muscular dystrophy (DMD): options by country

Sourced options by country plus visit-prep questions for Duchenne muscular dystrophy (DMD). Each line links to its regulator, HTA, or guideline source. This page maps options; it does not recommend or rank them.

Options mappedRare diseaseLast checked June 2026

What this page does

Maps options by country

It maps sourced options by country alongside diagnosis wording, stage, test results, specialists, and trial-search terms.

What it does not do

Does not choose treatment

It does not rank treatments, recommend a choice, or decide clinical fit.

Where it comes from

Built on trusted sources

Every option links to a trusted regulator, HTA, or guideline source, and the list grows as new sources pass verification.

Information to gather before the next visit

Which exact DMD mutation and exon-skipping amenability are documented?
Is the person ambulatory or non-ambulatory, and how does that affect gene-therapy availability?
Are corticosteroid, vamorolone, givinostat, exon-skipping, or gene therapy being discussed as separate options with different monitoring needs?
Is access being determined by EMA authorization, NICE appraisal, or another national payer?

Trial-search terms to discuss

Duchenne muscular dystrophy (DMD) clinical trial

Options by country

Treatments by country

Regulatory and access status by country, from official sources. It shows what exists and where — not a recommendation.

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.

Sources

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

This is official regulatory and access status only — not medical advice, not a recommendation, and not a statement about eligibility. Whether any option fits depends on your situation and your oncology team. Status changes over time; confirm the current position with the linked source. Last checked 2026-06-12.

Beyond approved care

In clinical trials & emerging options

Options that are not — or not yet — an approved standard where you live: studies, clinical trials, off-label use, and early evidence that your own oncologist may not raise. Each is labeled by how strong the evidence is. A listing here is information to research and discuss with your team; it does not mean a treatment is proven, safe for you, or available today.

In clinical trials

Vamorolone; prednisone comparator; idebenone; placeboClinical trial · NCT03439670Clinical trialTrial 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
Vamorolone; prednisone comparator; idebenone; placeboClinical trial · NCT03439670Clinical trialTrial 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
Vamorolone; prednisone comparator; idebenone; placeboClinical trial · NCT03439670Clinical trialTrial 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-06939926Clinical trial · NCT05524883Clinical trialTrial 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
DYNE-251; casimersen / SRP-4045; golodirsen / SRP-4053; viltolarsen; delandistrogene moxeparvovec / SRP-9001; PF-06939926Clinical trial · NCT05524883Clinical trialTrial 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
DYNE-251; casimersen / SRP-4045; golodirsen / SRP-4053; viltolarsen; delandistrogene moxeparvovec / SRP-9001; PF-06939926Clinical trial · NCT05524883Clinical trialTrial 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-3247Clinical trialClinical trialTrial 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
SAT-3247Clinical trialClinical trialTrial 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)Clinical trialClinical trialInvestigationalAustralia · 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)Clinical trialClinical trialInvestigationalAustralia · 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-5141bClinical trial · NCT03179631Clinical trialTrial 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
Ataluren / Translarna; NS-089/NCNP-02; DS-5141bClinical trial · NCT03179631Clinical trialTrial 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

A clinical-trial listing or early report shows an option is being studied — not that it works, that it is safe for any one person, or that a site is enrolling today. Whether any of these fits is a conversation for your oncology team and the trial team. Last checked 2026-06-12.