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找到 76 条关于
“argininosuccinic aciduria”
全部
76
临床试验
60
药物
8
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0
资讯
8
去年
Breakthrough Gene Editing Therapy Successfully Treats Infant with Rare Metabolic Disorder
2个月前
Genetic Study Reveals Elevated Risk of Rare Inherited Diseases in Scottish Traveller Founder Population
10个月前
Medunik USA Expands Medicaid Coverage for Pheburane in Rare Disease Treatment Access Initiative
2个月前
Hypoxia Therapy Dramatically Extends Lifespan in Mouse Model of HTRA2-Linked Mitochondrial Disease
去年
Genespire Secures $52 Million to Advance Gene Therapy for Methylmalonic Acidemia
去年
Genespire Presents Promising Dosing Data for First-in-Human Gene Therapy in Methylmalonic Acidemia
5个月前
FDA Clears IND for Gene Therapy Candidate MMA-101 to Treat Rare Metabolic Disorder Methylmalonic Acidemia
2个月前
Genespire and SR-TIGET Demonstrate Durable Preclinical Efficacy of Liver-Directed Gene Therapy for Methylmalonic Acidemia