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找到 386 条关于
“NPC1”
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386
临床试验
303
药物
55
机构
0
资讯
28
去年
Rafael Holdings Reports 78% Success Rate for NPC1 Treatment in Phase 3 Pediatric Sub-Study
去年
Trappsol Cyclo Shows Promise in Stabilizing or Improving Niemann-Pick Disease Type C1 in Young Patients
去年
Rafael Holdings Completes $25 Million Rights Offering to Fund Trappsol Cyclo Phase 3 Trial for Rare Disease
4个月前
Rafael Holdings Secures Exclusive MIT License for Cyclodextrin Therapy Targeting ApoE4-Positive Alzheimer's Patients
8天前
Beren Therapeutics and GeneDx Launch 'Test for NPC' to Accelerate Diagnosis of Niemann-Pick Disease Type C
2年前
FDA Advisory Committee Supports Arimoclomol for Niemann-Pick Disease Type C
2个月前
Nanobody-Based Bispecific Antibody Delivers Broad-Spectrum Protection Against Multiple Ebolavirus Species
去年
Azafaros' Nizubaglustat Gains Orphan Drug Designations, Phase 3 Trials to Start in 2025
去年
Zevra Therapeutics Launches MIPLYFFA, First FDA-Approved Treatment for Niemann-Pick Disease Type C
7个月前
Azafaros to Present Phase 3 Nizubaglustat Data for Rare Lysosomal Storage Disorders at WORLDSymposium 2026
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