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找到 8,624 条关于
“mucopolysaccharidosis IIIA”
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8,624
临床试验
7,393
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130
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4
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1,097
2年前
JCR Pharmaceuticals' JR-441 Receives Clearance for Phase I MPS IIIA Trial in Japan
3年前
Ultragenyx's UX111 Gene Therapy Shows Sustained Efficacy in MPS IIIA Patients
去年
Ultragenyx's UX111 Gene Therapy Shows Promise in Sanfilippo Syndrome Type A
去年
FDA's START Program Selects First Rare Disease Therapies for Accelerated Development
去年
2024 Breakthroughs and Setbacks in Lysosomal Disorder Gene Therapies
2年前
Ultragenyx to Pursue Accelerated Approval for UX111 Gene Therapy in MPSIIIA
4个月前
Orchard Therapeutics Receives Innovation Passport for Gene Therapy OTL-201 Targeting Rare Sanfilippo Syndrome
去年
Advancements in Gene and Cell Therapies Target Diverse Diseases
上个月
Shared Lysosomal Pathway Drives Neurodegeneration in Rare Childhood Dementia and Alzheimer's, UC San Diego Study Reveals
昨天
FDA Approves Fayuvi, First-Ever Gene Therapy for Sanfilippo Syndrome Type A
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