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找到 35,627 条关于
“neurometabolic disorder”
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35,627
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去年
FDA Approves Kebilidi, a Novel Gene Therapy for AADC Deficiency
去年
Breakthrough Discovery: PLA2G15 Identified as Novel Target for Neurometabolic Disorders
去年
Minoryx Initiates Phase 2a Trial of Leriglitazone for Rett Syndrome
4个月前
Orchard Therapeutics Receives Innovation Passport for Gene Therapy OTL-201 Targeting Rare Sanfilippo Syndrome
去年
Khondrion's Sonlicromanol Receives FDA Clearance for Phase 3 Trial in Primary Mitochondrial Disease
去年
Khondrion's Sonlicromanol Receives FDA Clearance for Phase 3 Trial in Primary Mitochondrial Disease
去年
Khondrion's Sonlicromanol Receives FDA Clearance for Phase 3 Trial in Primary Mitochondrial Disease
5个月前
Deep Learning and Brain Organoids Uncover Azole Antifungals as Repurposable Drug Candidates for Leigh Syndrome
3个月前
Ketogenic Therapy Shows Promise for Anorexia Nervosa Treatment in UC San Diego Clinical Trial
去年
Orchard Therapeutics' Lenmeldy Shows Long-Term Efficacy in Treating Metachromatic Leukodystrophy, NEJM Study Reveals
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