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Mucopolysaccharidosis (MPS): 국가별 선택지

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선택지 정리됨희귀·유전 질환최종 확인 2026.06

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임상시험 검색어

Mucopolysaccharidosis (MPS) 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

  • Laronidase (Aldurazyme); idursulfase (Elaprase)[1]FDA-approvedAlpha-L-iduronidase deficiency / IDUA mutation for MPS I; iduronate-2-sulfatase deficiency / IDS mutation for MPS II; Long-term enzyme replacement therapy for non-CNS/systemic MPS I or MPS II manifestations as described in labels. · Aldurazyme has not been evaluated for central nervous system manifestations. Elaprase label includes age/symptom caveats and infusion/anaphylaxis precautions. Hematopoietic stem-cell transplant and supportive care are not fully captured in this first pass. Confidence/conflicts: High for U.S. FDA-label indications; CNS/transplant/supportive pathways need deeper source work.
  • Laronidase (Aldurazyme); idursulfase (Elaprase)[1]FDA-approvedAlpha-L-iduronidase deficiency / IDUA mutation for MPS I; iduronate-2-sulfatase deficiency / IDS mutation for MPS II; Long-term enzyme replacement therapy for non-CNS/systemic MPS I or MPS II manifestations as described in labels. · Aldurazyme has not been evaluated for central nervous system manifestations. Elaprase label includes age/symptom caveats and infusion/anaphylaxis precautions. Hematopoietic stem-cell transplant and supportive care are not fully captured in this first pass. Confidence/conflicts: High for U.S. FDA-label indications; CNS/transplant/supportive pathways need deeper source work.
  • Laronidase (Aldurazyme); idursulfase (Elaprase)[1]FDA-approvedAlpha-L-iduronidase deficiency / IDUA mutation for MPS I; iduronate-2-sulfatase deficiency / IDS mutation for MPS II; Long-term enzyme replacement therapy for non-CNS/systemic MPS I or MPS II manifestations as described in labels. · Aldurazyme has not been evaluated for central nervous system manifestations. Elaprase label includes age/symptom caveats and infusion/anaphylaxis precautions. Hematopoietic stem-cell transplant and supportive care are not fully captured in this first pass. Confidence/conflicts: High for U.S. FDA-label indications; CNS/transplant/supportive pathways need deeper source work.

European Union

  • Aldurazyme; Elaprase; Vimizim; Mepsevii[2]EMA authorisedAlpha-L-iduronidase deficiency; iduronate-2-sulfatase deficiency; GALNS deficiency for MPS IVA; beta-glucuronidase deficiency for MPS VII; Long-term ERT by MPS subtype; MPS I source explicitly non-neurological manifestations. · EMA authorization does not equal member-state reimbursement. UK, France, and Germany access/reimbursement remain source-pending. MPS VII and MPS IVA entries are adjacent to Hurler/Hunter but included to avoid missing available MPS-category options. Confidence/conflicts: High for EMA authorizations; medium for UK detail pending final NICE subtype-by-subtype pass.
  • Aldurazyme; Elaprase; Vimizim; Mepsevii[2]EMA authorisedAlpha-L-iduronidase deficiency; iduronate-2-sulfatase deficiency; GALNS deficiency for MPS IVA; beta-glucuronidase deficiency for MPS VII; Long-term ERT by MPS subtype; MPS I source explicitly non-neurological manifestations. · EMA authorization does not equal member-state reimbursement. UK, France, and Germany access/reimbursement remain source-pending. MPS VII and MPS IVA entries are adjacent to Hurler/Hunter but included to avoid missing available MPS-category options. Confidence/conflicts: High for EMA authorizations; medium for UK detail pending final NICE subtype-by-subtype pass.

Japan

  • elosulfase alfa (Vimizim)[3]PMDA-approved (Japan)N-acetylgalactosamine-6-sulfatase / GALNS deficiency context; source names MPS IVA; enzyme replacement therapy for MPS IVA / Morquio A syndrome. · The fetched PMDA list verifies approval and indication but does not provide full label details, reimbursement, respiratory/orthopedic/anesthesia precautions, infusion-reaction management, or mobility endpoint limitations. Full current Japanese package insert remains a gap. Confidence/conflicts: High for Japanese Vimizim MPS IVA approval and indication from PMDA list; no conflict identified. Full label and reimbursement remain gaps.
  • idursulfase (Elaprase)[3]PMDA-approved (Japan)iduronate-2-sulfatase (IDS) deficiency context; X-linked recessive MPS II; intravenous enzyme replacement therapy for mucopolysaccharidosis II. · PMDA's Izcargo review report states idursulfase/Elaprase is currently available in Japan as intravenous enzyme replacement therapy for MPS II, but because IDS cannot cross the blood-brain barrier, intravenous Elaprase is unlikely to treat CNS symptoms. This entry does not establish reimbursement, long-term neurologic outcomes, infusion reaction protocols, or full current package insert details. Confidence/conflicts: High for Japanese Elaprase MPS II approval and CNS limitation context; no conflict identified. Full current label and reimbursement remain gaps.
  • pabinafusp alfa (Izcargo for I.V. Infusion)[4]PMDA-approved (Japan)iduronate-2-sulfatase (IDS) deficiency context; CNS symptoms/progression context emphasized in source; weekly intravenous treatment for MPS II; PMDA accepted a precaution that pabinafusp alfa should be administered to patients in whom improvement of CNS symptoms or suppression of CNS-symptom progression is considered necessary. · PMDA states long-term efficacy and other aspects should be further evaluated. The review notes antibody production and recommends regular anti-drug antibody testing, with close monitoring for infusion-associated reactions in antibody-positive patients. This entry does not establish reimbursement, comparative choice versus Elaprase, or individual eligibility. Confidence/conflicts: High for Japanese Izcargo MPS II approval and CNS-precaution context; no conflict identified. Long-term evidence and reimbursement remain gaps.

Australia

  • idursulfase (Elaprase)[5]Approvediduronate-2-sulfatase deficiency / IDS mutation context; Australian LSDP access for idursulfase in MPS II; long-term ERT for Hunter syndrome. · LSDP access is subject to guideline eligibility and reapplication requirements. Healthdirect reflects ARTG medicine information but is not a full TGA PI or payer rule. Confidence/conflicts: Medium-high; Australian access and ARTG-linked indication verified, but full current PI criteria remain source-pending. Availability/reimbursement outside the approving regulator not established.
  • idursulfase (Elaprase)[5]Approvediduronate-2-sulfatase deficiency / IDS mutation context; Australian LSDP access for idursulfase in MPS II; long-term ERT for Hunter syndrome. · LSDP access is subject to guideline eligibility and reapplication requirements. Healthdirect reflects ARTG medicine information but is not a full TGA PI or payer rule. Confidence/conflicts: Medium-high; Australian access and ARTG-linked indication verified, but full current PI criteria remain source-pending. Availability/reimbursement outside the approving regulator not established.
  • laronidase (Aldurazyme); idursulfase (Elaprase)[6]ApprovedMPS I alpha-L-iduronidase deficiency; MPS II iduronate-2-sulfatase deficiency; Australian LSDP-funded ERT access pathways for MPS I and MPS II. · The general LSDP page identifies funded medicines and conditions but not full clinical criteria. Subtype-specific guidelines and current PI should be checked before reuse. Confidence/conflicts: Medium-high for Australian LSDP medicine-condition mapping; detailed MPS I guideline extraction remains a follow-up. Availability/reimbursement outside the approving regulator not established.
  • laronidase (Aldurazyme); idursulfase (Elaprase)[6]ApprovedMPS I alpha-L-iduronidase deficiency; MPS II iduronate-2-sulfatase deficiency; Australian LSDP-funded ERT access pathways for MPS I and MPS II. · The general LSDP page identifies funded medicines and conditions but not full clinical criteria. Subtype-specific guidelines and current PI should be checked before reuse. Confidence/conflicts: Medium-high for Australian LSDP medicine-condition mapping; detailed MPS I guideline extraction remains a follow-up. Availability/reimbursement outside the approving regulator not established.

Thailand

  • idursulfase (Elaprase)[7]Approvediduronate-2-sulfatase deficiency / IDS-related MPS II context; severe mutation status relevant for hypersensitivity/antibody risk per source; enzyme replacement therapy for Hunter syndrome/MPS II; source specifies evidence distinctions by age group. · Safety and efficacy are not established in pediatric patients younger than 16 months. The label includes serious hypersensitivity/anaphylaxis, respiratory/cardiac monitoring, and antibody-risk cautions, including higher risks in patients with complete gene deletion, large rearrangement, nonsense, frameshift, or splice-site mutations. This entry does not establish reimbursement or center availability in Thailand. Confidence/conflicts: High for Thai Elaprase label and authorization details; no conflict identified, but reimbursement/access pathway remains unresolved.

Canada

  • idursulfase (Elaprase)[8]Health Canada approvediduronate-2-sulfatase deficiency; long-term ERT for MPS II/Hunter syndrome in Canada. · The product monograph verifies label status but not public-plan coverage. It notes supplemental oxygen should be available during infusion for patients who use oxygen if an infusion-related reaction occurs. Confidence/conflicts: High for Canadian Elaprase label and marketed status; no conflict identified. Availability/reimbursement outside the approving regulator not established.
  • idursulfase (Elaprase)[8]Health Canada approvediduronate-2-sulfatase deficiency; long-term ERT for MPS II/Hunter syndrome in Canada. · The product monograph verifies label status but not public-plan coverage. It notes supplemental oxygen should be available during infusion for patients who use oxygen if an infusion-related reaction occurs. Confidence/conflicts: High for Canadian Elaprase label and marketed status; no conflict identified. Availability/reimbursement outside the approving regulator not established.
  • laronidase (Aldurazyme)[9]Approvedalpha-L-iduronidase deficiency; long-term ERT for non-CNS manifestations of MPS I in Canada. · Aldurazyme does not address central nervous system manifestations in the label wording. The monograph highlights life-threatening anaphylactic reactions and need for appropriate medical support during and after infusion. Confidence/conflicts: High for Canadian label; reimbursement/HSCT pathway remains source-pending.
  • laronidase (Aldurazyme)[9]Approvedalpha-L-iduronidase deficiency; long-term ERT for non-CNS manifestations of MPS I in Canada. · Aldurazyme does not address central nervous system manifestations in the label wording. The monograph highlights life-threatening anaphylactic reactions and need for appropriate medical support during and after infusion. Confidence/conflicts: High for Canadian label; reimbursement/HSCT pathway remains source-pending.

출처

  1. U.S. Food and Drug Administration — official drug label · official drug label
  2. European Medicines Agency (EMA) — regulator EPAR · regulator EPAR
  3. Pharmaceuticals and Medical Devices Agency (PMDA) — approved drugs list PDF · approved drugs list PDF
  4. Pharmaceuticals and Medical Devices Agency (PMDA) — regulator deliberation/review report PDF · regulator deliberation/review report PDF
  5. Australian Government Department of Health, Disability and Ageing — LSDP resource collection · LSDP resource collection
  6. Australian Government Department of Health, Disability and Ageing — LSDP program description · LSDP program description
  7. Thai National Drug Information / Thai FDA-MOPH — SmPC PDF / regulator drug-information repository · SmPC PDF / regulator drug-information repository
  8. Health Canada product monograph repository / Takeda Canada — official product monograph · official product monograph
  9. Sanofi-aventis Canada / Health Canada product monograph — 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. 임상 세부 내용은 영문이 정본입니다. 최종 확인 2026-06-12.