Arimoclomol in combination with miglustat for use in the treatment of lysosomal storage disorders
Arimoclomol, combined with miglustat, addresses the lack of disease-modifying therapies for NPC by activating TFEB/TFE3 to upregulate NPC1, effectively reducing cholesterol and slowing disease progression in NPC patients.
WO2026010892A1PCT designated stage Publication Date: 2026-01-08ZEVRA THERAPEUTICS INC
Patent Information
- Application Number
- PCT/US2025/035980
- Authority / Receiving Office
- WO · WO
- Patent Type
- Applications
- Current Assignee / Owner
- Priority Date
- 2025-06-30
- Filing Date
- 2025-06-30
- Publication Date
- 2026-01-08
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Figure US2025035980_08012026_PF_FP_ABST
Abstract
The present disclosure provides methods of increasing removal of lysosomal cholesterol in a patient in need thereof by administering a therapeutically effective amount of arimoclomol in combination with miglustat. The present disclosure further provides methos of reducing and / or delaying Niemann-Pick Disease, Type-C, progression in said patient.
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Citation Information
Patent Citations
Arimoclomol for the treatment of niemann pick disease, type c, in patients with er type missense mutations
WO2022136640A1
Arimoclomol compositions for use in treating niemann pick disease type c (NPC)
WO2024089667A1