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找到 90,406 条关于
“ultrarare disease”
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去年
FDA Raises Doubts About Stealth BioTherapeutics' Elamipretide for Barth Syndrome
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FDA Approves PTC Therapeutics' Kebilidi, First Direct-to-Brain Gene Therapy
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FDA Authorizes Expanded Access Programs for Jaguar Health's Crofelemer in Rare Pediatric Intestinal Disease
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Ultragenyx Reports Positive Q3 2024 Results and Clinical Updates
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Jaguar Health Advances Crofelemer Trials for Rare Diarrheal Diseases
去年
Pharmacists Lead Implementation of Advanced Cell and Gene Therapies Across Major Health Systems
6个月前
Progeria Research Foundation Partners with Forge Biologics to Advance CRISPR Gene Therapy for Rare Aging Disease
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