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找到 78 条关于
“Metachromatic Leukodystrophy”
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78
临床试验
53
药物
4
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0
资讯
21
去年
Orchard Therapeutics' Lenmeldy Shows Long-Term Efficacy in Treating Metachromatic Leukodystrophy, NEJM Study Reveals
去年
FDA Approves Lenmeldy: $4.25 Million Gene Therapy for Rare Childhood Disease MLD
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
Neuroscience Drug Development: A Year of Highs and Lows in 2024
10个月前
Japan Grants Orphan Designation to OTL-200 Gene Therapy for Rare Childhood Disease MLD
去年
NHS England Launches Groundbreaking Newborn Screening for 200+ Genetic Conditions
3个月前
Right to Try for Individualized Treatments Act Introduced in Congress to Expand Access to Gene Therapies for Rare Disease Patients
去年
FDA Approves Novel Therapies for Frostbite, Food Allergies, and Synovial Sarcoma in 2024
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