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找到 32,445 条关于
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11天前
Global Genes Partners with Citizen Health to Power RARE-X Rare Disease Data Platform
去年
Researchers Identify Two Drug Candidates for Ultra-Rare X-Linked Myopathy Using Zebrafish Model
2年前
FDA Grants Priority Review for CUTX-101 in Treatment of Menkes Disease
去年
Egetis Therapeutics' Emcitate Poised for European Approval, Driving Optimistic Financial Outlook
去年
Chiesi's ELFABRIO Shows Promise in Phase 3 Trial for Fabry Disease with Twice-Dose Regimen
2年前
CRISPR-Cas9 Corrects Dystrophin Duplications in DMD Patient Cells
去年
Gene Therapy Skysona Linked to Hematological Cancers in CALD Patients
去年
Elevidys Fails to Meet Primary Endpoint in Phase 3 EMBARK Trial for Duchenne Muscular Dystrophy
去年
AceLink Therapeutics' AL01211 Shows Promising Safety and Biomarker Reduction in Phase 2 Fabry Disease Trial
2个月前
New International Guideline Published for Diagnosing and Treating Allan-Herndon-Dudley Syndrome
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