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A total of 549 posts are filed under Innovation
FDA Approves Leadiant Biosciences’ Enzyme Replacement Therapy for ADA-SCID
Rare Daily Staff The U.S. Food and Drug Administration approved Revcovi, Leadiant Biosciences’ enzyme replacement therapy for the… Continue Reading
EMA Grants PRIME Designation to Orchard Therapeutics’ Beta-Thalassemia Gene Therapy
Rare Daily Staff The European Medicines Agency granted Orchard Therapeutics Priority Medicines (PRIME) designation to OTL-300, an… Continue Reading
Discovery Could Lead to Better Diagnosis and Treatment of Lupus in African Patients
Rare Daily Staff Black Africans have a higher level of Lupus than previous known and identified an antibody previously unlinked to the… Continue Reading
NIH Expands Undiagnosed Diseases Network with $100 Million in New Grants
Rare Daily Staff The National Institutes of Health said it would provide about $100 million in grants over the next four years to expand its… Continue Reading
Amicus Therapeutics to Acquire Celenex, Scoring Gene Therapy Portfolio for Neurologic Disorders
Rare Daily Staff Amicus Therapeutics agreed to acquire Celenex in a deal that provides it worldwide development and commercial rights for… Continue Reading
FDA Grants Cellectar Biosciences Rare Pediatric Disease Designation for Experimental Osteosarcoma Drug
Rare Daily Staff The U. S. Food and Drug Administration has granted Rare Pediatric Disease designation to Cellectar Biosciences for its… Continue Reading
Thinking Ahead
When the European Medicines Agency approved Myozyme in 2006, Genzyme’s enzyme replacement therapy for the lysosomal storage disorder… Continue Reading

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