hemophilia A – media

Hemophilia A Gene Therapy by Spark Therapeutics Needs Phase 3 Trial

People with the bleeding disorder hemophilia A require lifelong treatment, which would include regular infusions of a clotting promoter factor VIII. Treatments could be expensive and time-consuming.
- Source: FierceBiotech
- 838
- August 9, 2018
Clinical Trials Gene therapy hemophilia A
FDA Calls BioMarin’s Hemophilia A Drug a Breakthrough

BioMarin Pharmaceutical announced that the U.S. Food and Drug Administration (FDA) granted its valoctocogene roxaparvovec for Hemophilia A Breakthrough Therapy Designation.
- Source: biospace
- 815
- October 31, 2017
Clinical Trial FDA hemophilia A
Shire Receives Orphan Drug Designation for Gene Therapy Candidate SHP654 (BAX 888) for the Treatment of Hemophilia A

Shire plc (LSE: SHP, NASDAQ: SHPG), the global biotechnology leader in rare diseases, announced the U.S. Food and Drug Administration (FDA) awarded Orphan Drug Designation(ODD) to Shire`s gene therapy candidate SHP654 (also designated as BAX 888), an investigational factor VIII (FVIII) gene therapy for the treatment of hemophilia A. The regulatory agency also granted Shire investigational new drug (IND) status for SHP654.
- Source: finance.yahoo
- 469
- October 27, 2017
FDA hemophilia A Orphan Drug Designation Shire

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