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找到 101,463 条关于
“lysosomal storage disease”
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101,463
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10个月前
Polaryx Therapeutics Secures Nasdaq Listing Ahead of Phase 2 Trial for Rare Lysosomal Storage Disorders
2年前
GC Biopharma and Hanmi Pharmaceutical's Fabry Disease Treatment LA-GLA Receives FDA IND Clearance
7年前
Denali's Hunter Syndrome Candidate Receives FDA Orphan Drug and Rare Pediatric Disease Designations
去年
Ultragenyx's UX111 Gene Therapy for Sanfilippo Syndrome Type A Receives FDA Priority Review
去年
Ultragenyx's UX111 Gene Therapy Shows Promise in Sanfilippo Syndrome Type A
上个月
Malaysia Registers 26 Orphan Medicines Since 2020 as Rare Disease List Reaches 529 Conditions
去年
Immusoft's Engineered B-Cell Therapy Shows Promise in MPS I Trial
去年
Azafaros Secures €130M Funding to Advance Nizubaglustat into Phase III for Rare Lysosomal Storage Disorders
去年
FDA Approves Five Groundbreaking Treatments for Ultra-Rare Diseases in Recent Months
去年
FDA Issues Complete Response Letter for Ultragenyx's UX111 Gene Therapy for Sanfilippo Syndrome Type A
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