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Spinal muscular atrophy (SMA): options by country

Sourced options by country plus visit-prep questions for Spinal muscular atrophy (SMA). 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 SMN1 and SMN2 genetic results are documented?
  • Is the option an SMN2 splicing therapy, IV gene therapy, or intrathecal gene therapy?
  • Do age, prior treatment, anti-AAV9 antibodies, liver status, or ventilation status affect the local plan?
  • Is access being determined by EMA authorization, NICE appraisal, or another national payer?

Trial-search terms to discuss

Spinal muscular atrophy (SMA) 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

  • Nusinersen / Spinraza; risdiplam / Evrysdi; onasemnogene abeparvovec-xioi / Zolgensma; onasemnogene abeparvovec-brve / Itvisma[1]FDA-approvedSMN1 mutation or bi-allelic SMN1 mutation for gene therapy; SMN2 copy and age restrictions vary by product/source; Pediatric and adult SMA for nusinersen; patients 2 months and older for risdiplam; pediatric patients younger than 2 years for IV Zolgensma; adult and pediatric patients aged 2 years and older for intrathecal Itvisma. · Gene-therapy products have age, mutation, administration-route, and safety-monitoring requirements. FDA sources do not determine payer coverage or individual eligibility. Confidence/conflicts: High for U.S. approval status and label-scope summaries. No source conflict identified.
  • Nusinersen / Spinraza; risdiplam / Evrysdi; onasemnogene abeparvovec-xioi / Zolgensma; onasemnogene abeparvovec-brve / Itvisma[1]FDA-approvedSMN1 mutation or bi-allelic SMN1 mutation for gene therapy; SMN2 copy and age restrictions vary by product/source; Pediatric and adult SMA for nusinersen; patients 2 months and older for risdiplam; pediatric patients younger than 2 years for IV Zolgensma; adult and pediatric patients aged 2 years and older for intrathecal Itvisma. · Gene-therapy products have age, mutation, administration-route, and safety-monitoring requirements. FDA sources do not determine payer coverage or individual eligibility. Confidence/conflicts: High for U.S. approval status and label-scope summaries. No source conflict identified.

European Union

  • Nusinersen / Spinraza; risdiplam / Evrysdi; onasemnogene abeparvovec / Zolgensma[2]Approved (restricted indication)5q SMA; SMN1 inherited mutations for Zolgensma; SMN2 copy/clinical type restrictions per EMA product pages; EMA-labeled disease-modifying therapy for 5q SMA; NHS routine-access and presymptomatic gene-therapy appraisal contexts as specified by NICE. · EMA authorization does not establish price/reimbursement in every EU member state. NICE final draft guidance and HST guidance apply to England/NHS appraisal scope and are not identical to EU or devolved-country access. Confidence/conflicts: High for EMA and NICE statements; no conflict identified, but NICE scope is national and conditional by recommendation wording.
  • Nusinersen / Spinraza; risdiplam / Evrysdi; onasemnogene abeparvovec / Zolgensma[2]Approved (restricted indication)5q SMA; SMN1 inherited mutations for Zolgensma; SMN2 copy/clinical type restrictions per EMA product pages; EMA-labeled disease-modifying therapy for 5q SMA; NHS routine-access and presymptomatic gene-therapy appraisal contexts as specified by NICE. · EMA authorization does not establish price/reimbursement in every EU member state. NICE final draft guidance and HST guidance apply to England/NHS appraisal scope and are not identical to EU or devolved-country access. Confidence/conflicts: High for EMA and NICE statements; no conflict identified, but NICE scope is national and conditional by recommendation wording.

Japan

  • Onasemnogene abeparvovec / Zolgensma; risdiplam / Evrysdi; nusinersen noted in PMDA review context[3]PMDA-approved (Japan)Bi-allelic SMN1 deletion or mutation for Zolgensma; anti-AAV9 antibody negative condition; SMA indication for risdiplam; Zolgensma in symptomatic or presymptomatic SMA patients younger than 2 years with bi-allelic SMN1 mutation/deletion and anti-AAV9 antibody criteria; Evrysdi for SMA across infants, children, and adults as described by Chugai announcement. · Novartis and Chugai announcements are manufacturer sources; PMDA report is authoritative for Zolgensma. Primary PMDA risdiplam label remains an active gap. Confidence/conflicts: High for Japan Zolgensma via PMDA; medium-high for Evrysdi because source is manufacturer announcement naming MHLW approval. Availability/reimbursement outside the approving regulator not established.
  • Onasemnogene abeparvovec / Zolgensma; risdiplam / Evrysdi; nusinersen noted in PMDA review context[3]PMDA-approved (Japan)Bi-allelic SMN1 deletion or mutation for Zolgensma; anti-AAV9 antibody negative condition; SMA indication for risdiplam; Zolgensma in symptomatic or presymptomatic SMA patients younger than 2 years with bi-allelic SMN1 mutation/deletion and anti-AAV9 antibody criteria; Evrysdi for SMA across infants, children, and adults as described by Chugai announcement. · Novartis and Chugai announcements are manufacturer sources; PMDA report is authoritative for Zolgensma. Primary PMDA risdiplam label remains an active gap. Confidence/conflicts: High for Japan Zolgensma via PMDA; medium-high for Evrysdi because source is manufacturer announcement naming MHLW approval. Availability/reimbursement outside the approving regulator not established.

Australia

  • nusinersen (Spinraza)[4]TGA-registered (Australia)5q SMA / SMN1-related disease context; SMN2-targeted treatment mechanism; 5q SMA treatment in Australia; intrathecal therapy context should be checked against current Australian product information and payer criteria. · AusPARs are static documents tied to a submission at a particular point in time. This entry records regulator assessment context, not current PBS criteria, intrathecal procedural suitability, or individual eligibility. Confidence/conflicts: Medium-high for Australian nusinersen regulatory context; current PI/PBS implementation remains a follow-up. Availability/reimbursement outside the approving regulator not established.
  • nusinersen (Spinraza)[4]TGA-registered (Australia)5q SMA / SMN1-related disease context; SMN2-targeted treatment mechanism; 5q SMA treatment in Australia; intrathecal therapy context should be checked against current Australian product information and payer criteria. · AusPARs are static documents tied to a submission at a particular point in time. This entry records regulator assessment context, not current PBS criteria, intrathecal procedural suitability, or individual eligibility. Confidence/conflicts: Medium-high for Australian nusinersen regulatory context; current PI/PBS implementation remains a follow-up. Availability/reimbursement outside the approving regulator not established.
  • nusinersen (Spinraza)[4]TGA-registered (Australia)5q SMA / SMN1-related disease context; SMN2-targeted treatment mechanism; 5q SMA treatment in Australia; intrathecal therapy context should be checked against current Australian product information and payer criteria. · AusPARs are static documents tied to a submission at a particular point in time. This entry records regulator assessment context, not current PBS criteria, intrathecal procedural suitability, or individual eligibility. Confidence/conflicts: Medium-high for Australian nusinersen regulatory context; current PI/PBS implementation remains a follow-up. Availability/reimbursement outside the approving regulator not established.
  • risdiplam (Evrysdi)[5]TGA-registered (Australia)5q SMA / SMN2 splicing modifier context; 5q SMA in patients aged 2 months and older in Australia. · The TGA page records regulatory status and mechanism context, not PBS subsidy, newborn-screening pathway, or individual suitability. Confidence/conflicts: High for Australian Evrysdi regulatory status; no conflict identified. Availability/reimbursement outside the approving regulator not established.
  • risdiplam (Evrysdi)[5]TGA-registered (Australia)5q SMA / SMN2 splicing modifier context; 5q SMA in patients aged 2 months and older in Australia. · The TGA page records regulatory status and mechanism context, not PBS subsidy, newborn-screening pathway, or individual suitability. Confidence/conflicts: High for Australian Evrysdi regulatory status; no conflict identified. Availability/reimbursement outside the approving regulator not established.

Canada

  • nusinersen (Spinraza)[6]Health Canada approved5q SMA / SMN2-targeted antisense oligonucleotide context; Canadian 5q SMA treatment context, including pediatric evidence; monograph notes limited data over age 18. · The monograph states overall management still requires strategies for motor decline, respiratory complications, and feeding difficulties. It specifies intrathecal lumbar-puncture administration by experienced professionals. Confidence/conflicts: High for Canadian Spinraza label/regulatory context; no conflict identified. Availability/reimbursement outside the approving regulator not established.
  • nusinersen (Spinraza)[6]Health Canada approved5q SMA / SMN2-targeted antisense oligonucleotide context; Canadian 5q SMA treatment context, including pediatric evidence; monograph notes limited data over age 18. · The monograph states overall management still requires strategies for motor decline, respiratory complications, and feeding difficulties. It specifies intrathecal lumbar-puncture administration by experienced professionals. Confidence/conflicts: High for Canadian Spinraza label/regulatory context; no conflict identified. Availability/reimbursement outside the approving regulator not established.
  • nusinersen (Spinraza)[6]Health Canada approved5q SMA / SMN2-targeted antisense oligonucleotide context; Canadian 5q SMA treatment context, including pediatric evidence; monograph notes limited data over age 18. · The monograph states overall management still requires strategies for motor decline, respiratory complications, and feeding difficulties. It specifies intrathecal lumbar-puncture administration by experienced professionals. Confidence/conflicts: High for Canadian Spinraza label/regulatory context; no conflict identified. Availability/reimbursement outside the approving regulator not established.
  • risdiplam (Evrysdi)[7]Health Canada approvedSMN1/SMN2 disease mechanism context; Canadian SMA treatment context; Health Canada SBD specifies patients 2 months and older. · The product monograph includes oral/enteral formulation details and notes limited data in older adults; this entry does not establish payer coverage or choice among SMA disease-modifying therapies. Confidence/conflicts: High for Canadian Evrysdi label/regulatory context; no conflict identified. Availability/reimbursement outside the approving regulator not established.
  • risdiplam (Evrysdi)[7]Health Canada approvedSMN1/SMN2 disease mechanism context; Canadian SMA treatment context; Health Canada SBD specifies patients 2 months and older. · The product monograph includes oral/enteral formulation details and notes limited data in older adults; this entry does not establish payer coverage or choice among SMA disease-modifying therapies. Confidence/conflicts: High for Canadian Evrysdi label/regulatory context; no conflict identified. Availability/reimbursement outside the approving regulator not established.

Sources

  1. U.S. Food and Drug Administration — regulator approval notice · regulator approval notice
  2. European Medicines Agency (EMA) — regulator EPAR · regulator EPAR
  3. Pharmaceuticals and Medical Devices Agency / MHLW — regulator review report · regulator review report
  4. Therapeutic Goods Administration (TGA) — Australian Public Assessment Report · Australian Public Assessment Report
  5. Therapeutic Goods Administration (TGA) — Australian Prescription Medicine Decision Summary · Australian Prescription Medicine Decision Summary
  6. Biogen Canada / Health Canada product monograph format — official product monograph · official product monograph
  7. Hoffmann-La Roche Canada / Health Canada product monograph format — official product monograph · official product monograph

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

  • Nusinersen sodium injection / SpinrazaClinical trial · NCT04317794Clinical trialTrial only (registry)Korea · Genetic documentation of 5q-linked SMA or 5q SMA for nusinersen surveillance records; Commercial/postmarketing setting for patients receiving or newly prescribed nusinersen sodium injection. · Registry records are postmarketing studies, not the official regulator labels. Country reimbursement and prescribing criteria are not established by the registry. Confidence/conflicts: High for postmarketing/commercial-use registry cells; MFDS and NMPA primary labels remain source-pending. ClinicalTrials.gov — postmarketing surveillance registry
  • Nusinersen sodium injection / SpinrazaClinical trial · NCT04317794Clinical trialTrial only (registry)Korea · Genetic documentation of 5q-linked SMA or 5q SMA for nusinersen surveillance records; Commercial/postmarketing setting for patients receiving or newly prescribed nusinersen sodium injection. · Registry records are postmarketing studies, not the official regulator labels. Country reimbursement and prescribing criteria are not established by the registry. Confidence/conflicts: High for postmarketing/commercial-use registry cells; MFDS and NMPA primary labels remain source-pending. ClinicalTrials.gov — postmarketing surveillance registry
  • EXG001-307 injection; vesemnogene lantuparvovec / AAV-hSMN1; OAV101 / onasemnogene abeparvovec trial and long-term follow-upClinical trial · NCT06576388Clinical trialTrial only (registry)China · Bi-allelic SMN1 mutation/deletion or SMN1 mutation analysis; SMN2 copy criteria vary by study; anti-AAV9 antibody exclusion in vesemnogene lantuparvovec and OAV101 records; Type I SMA single-IV gene therapy study in infants; progressive SMA AAV-hSMN1 study; treatment-naive type 2 SMA ages 2 to under 18 years for intrathecal OAV101 trial; long-term follow-up after OAV101 IT or IV clinical-trial treatment. · Trial statuses include completed, recruiting, and follow-up. OAV101 long-term follow-up is for participants from prior OAV101 trials, not open routine access. Confidence/conflicts: High for registry-listed gene-therapy trial/follow-up cells; no approval claim is made for investigational products. ClinicalTrials.gov — clinical-trial registry
  • EXG001-307 injection; vesemnogene lantuparvovec / AAV-hSMN1; OAV101 / onasemnogene abeparvovec trial and long-term follow-upClinical trial · NCT06576388Clinical trialTrial only (registry)China · Bi-allelic SMN1 mutation/deletion or SMN1 mutation analysis; SMN2 copy criteria vary by study; anti-AAV9 antibody exclusion in vesemnogene lantuparvovec and OAV101 records; Type I SMA single-IV gene therapy study in infants; progressive SMA AAV-hSMN1 study; treatment-naive type 2 SMA ages 2 to under 18 years for intrathecal OAV101 trial; long-term follow-up after OAV101 IT or IV clinical-trial treatment. · Trial statuses include completed, recruiting, and follow-up. OAV101 long-term follow-up is for participants from prior OAV101 trials, not open routine access. Confidence/conflicts: High for registry-listed gene-therapy trial/follow-up cells; no approval claim is made for investigational products. ClinicalTrials.gov — clinical-trial registry
  • Risdiplam / Evrysdi; nusinersen / Spinraza higher-dose regimens; apitegromab adjunct to nusinersen or risdiplam; ANB-004 gene therapyClinical trial · NCT02913482Clinical trialTrial only (registry)Russia · 5q-autosomal recessive SMA; SMN1 loss-of-function/deletion or mutation; SMN2 copy criteria in infant/presymptomatic/gene-therapy records; Infant type 1 SMA for FIREFISH; presymptomatic infants for RAINBOWFISH; higher-dose nusinersen studies for infantile/later-onset and prior risdiplam-treated SMA; later-onset SMA receiving background nusinersen or risdiplam for apitegromab; young children with SMA for ANB-004. · Some studies are completed or active-not-recruiting. Apitegromab and ANB-004 remain investigational in the fetched records. Registry country listings do not equal approval. Confidence/conflicts: High for registry cells; no country regulator approval claim is made from trial records. ClinicalTrials.gov — clinical-trial registry
  • Risdiplam / Evrysdi; nusinersen / Spinraza higher-dose regimens; apitegromab adjunct to nusinersen or risdiplam; ANB-004 gene therapyClinical trial · NCT02913482Clinical trialTrial only (registry)Russia · 5q-autosomal recessive SMA; SMN1 loss-of-function/deletion or mutation; SMN2 copy criteria in infant/presymptomatic/gene-therapy records; Infant type 1 SMA for FIREFISH; presymptomatic infants for RAINBOWFISH; higher-dose nusinersen studies for infantile/later-onset and prior risdiplam-treated SMA; later-onset SMA receiving background nusinersen or risdiplam for apitegromab; young children with SMA for ANB-004. · Some studies are completed or active-not-recruiting. Apitegromab and ANB-004 remain investigational in the fetched records. Registry country listings do not equal approval. Confidence/conflicts: High for registry cells; no country regulator approval claim is made from trial records. ClinicalTrials.gov — clinical-trial registry
  • Risdiplam / Evrysdi; nusinersen / Spinraza higher-dose regimens; apitegromab adjunct to nusinersen or risdiplam; ANB-004 gene therapyClinical trial · NCT02913482Clinical trialTrial only (registry)Russia · 5q-autosomal recessive SMA; SMN1 loss-of-function/deletion or mutation; SMN2 copy criteria in infant/presymptomatic/gene-therapy records; Infant type 1 SMA for FIREFISH; presymptomatic infants for RAINBOWFISH; higher-dose nusinersen studies for infantile/later-onset and prior risdiplam-treated SMA; later-onset SMA receiving background nusinersen or risdiplam for apitegromab; young children with SMA for ANB-004. · Some studies are completed or active-not-recruiting. Apitegromab and ANB-004 remain investigational in the fetched records. Registry country listings do not equal approval. Confidence/conflicts: High for registry cells; no country regulator approval claim is made from trial records. 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.