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找到 536,263 条关于
“inborn errors of metabolism”
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7天前
Alltrna Doses First tRNA Therapy in Humans as AP003 Phase 1 Begins in Australia
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Rafael Holdings Reports 78% Success Rate for NPC1 Treatment in Phase 3 Pediatric Sub-Study
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mRNA Delivery to CD34+ Cells via Lipid Nanoparticles Shows Promise in Rhesus Monkeys
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Garuda Therapeutics Names Avanish Vellanki as New CEO to Advance Off-the-Shelf Stem Cell Platform
2年前
Denali Therapeutics Plans Accelerated Approval Filing for Tividenofusp Alfa in MPS II
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FDA Issues Complete Response Letter for Saol's SL1009 Treatment for Rare Pediatric Mitochondrial Disease
2年前
RGX-121 Gene Therapy Shows Sustained Efficacy in Hunter Syndrome Trial
25天前
PTC Therapeutics' Sephience (sepiapterin) Data Reinforce Broad PKU Benefits, Including Diet Liberalization and Sustained Metabolic Control
去年
BioMarin Reports Positive Phase 3 Results for PALYNZIQ in Adolescent PKU Patients
去年
Applied Therapeutics' Govorestat Awaits Potential FDA Approval for Classic Galactosemia
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