선택지 정리됨
척수성 근위축증(SMA): 국가별 치료 선택지
이 페이지는 한국어 임상 치료 설명을 새로 쓰지 않습니다. 권위 출처, 진료 전 정리 질문, 임상시험 검색어, 구축 상태를 보여주는 안전한 시작점입니다.
선택지 정리됨희귀·유전 질환최종 확인 2026.06
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
- 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 NICE final draft guidance and HST guidance apply to England/NHS appraisal scope and are not identical to EU or devolved-country access.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.
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.
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.
- 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.
- 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.
출처
- U.S. Food and Drug Administration — regulator approval notice · regulator approval notice
- European Medicines Agency (EMA) — regulator EPAR · regulator EPAR
- Pharmaceuticals and Medical Devices Agency / MHLW — regulator review report · regulator review report
- Therapeutic Goods Administration (TGA) — Australian Public Assessment Report · Australian Public Assessment Report
- Therapeutic Goods Administration (TGA) — Australian Prescription Medicine Decision Summary · Australian Prescription Medicine Decision Summary
- Biogen Canada / Health Canada product monograph format — official product monograph · official product monograph
- Hoffmann-La Roche Canada / Health Canada product monograph format — official product monograph · official product monograph
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. 일부 선택지는 신속·조건부 승인 상태로, 적응증이 축소되거나 철회될 수 있습니다. 임상 세부 내용은 영문이 정본입니다. 최종 확인 2026.06.
승인된 치료 너머
임상시험 및 신흥 선택지
환자가 거주하는 국가에서 아직 승인된 표준 치료가 아닌 선택지입니다 — 연구, 임상시험, 허가 외 사용, 담당 종양내과 의사가 먼저 언급하지 않을 수 있는 초기 근거입니다. 각 항목은 근거의 강도에 따라 구분됩니다. 여기에 실린 항목은 조사하고 의료진과 상의할 정보일 뿐, 효과가 입증되었거나 안전하거나 현재 이용 가능하다는 의미가 아닙니다. 임상 세부 내용은 영문이 정본입니다.
임상시험 진행 중
- Nusinersen sodium injection / Spinraza임상시험 · NCT04317794임상시험Trial 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-up임상시험 · NCT06576388임상시험Trial 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 therapy임상시험 · NCT02913482임상시험Trial 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
임상시험이나 초기 보고에 실렸다는 것은 해당 선택지가 연구되고 있다는 의미일 뿐, 효과가 있거나 특정 환자에게 안전하거나 현재 등록 가능하다는 뜻은 아닙니다. 어떤 선택지가 적합한지는 담당 종양내과 팀과 임상시험 팀이 함께 상의할 문제입니다. 최종 확인 2026.06.
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출처
공신력 있는 출처 기반
모든 선택지는 규제·평가·지침 등 공신력 있는 출처에 연결되며, 새 출처가 검증되면 계속 확장됩니다.
진료 전 정리할 정보
- 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?