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找到 83 条关于
“TUBB4A leukodystrophy”
全部
83
临床试验
53
药物
7
机构
2
资讯
21
2年前
SynaptixBio Secures Second FDA Orphan Drug Designation for TUBB4A Leukodystrophy Variant
去年
SynaptixBio Selects Antisense Oligonucleotide Candidate for Rare Disease H-ABC Clinical Trials
去年
Orchard Therapeutics' Lenmeldy Shows Long-Term Efficacy in Treating Metachromatic Leukodystrophy, NEJM Study Reveals
去年
SETU Secures €159,589 Grant for Novel Gene Therapy Research in Fatal Childhood Canavan Disease
去年
FDA Approves Lenmeldy: $4.25 Million Gene Therapy for Rare Childhood Disease MLD
上个月
Breakthrough trial offers first hope for children with Vanishing White Matter disease
2年前
Orchard Therapeutics' Gene Therapy Lenmeldy Approved for MLD, Priced at $4.25 Million
去年
FDA Approves Five Groundbreaking Treatments for Ultra-Rare Diseases in Recent Months
去年
U.S. Drug Prices Double as Pharmaceutical Industry Shifts Focus to Rare Diseases
去年
FDA Grants Fast Track Designation to Zilganersen for Alexander Disease
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