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Centessa Pharmaceuticals (CNTA) Announces Orphan Drug Designation Granted to SerpinPC for the Treatment of Hemophilia B

Centessa Pharmaceuticals (CNTA) Announces Orphan Drug Designation Granted to SerpinPC for the Treatment of Hemophilia B
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Saurabh sahaDrug administrationResearch developmentCentessa pharmaceuticalsOrphan drug designationAnnualized bleeding rateChief executive officerDrug designation

BioMarin Announces Publication in New England Journal of Medicine of One-Year Results from Phase 3 Pivotal Trial with Valoctocogene Roxaparvovec Gene Therapy in Adults with Severe Hemophilia A

/PRNewswire/ BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced publication of results from the Phase 3 GENEr8-1 study of valoctocogene.

Debra charlesworthTraci mccartyExchange commissionBiomarin pharmaceutical incData monitoring committeeNew england journalMedicine publishes editorialReduced bleedingBaseline factorAnnualized bleeding rateRoxaparvovec gene therapyBiomarin pharmaceuticalMarin pharmaceuticalBiomarin pharmaceutical inc

BioMarin Announces Oral Presentation of 2-Year Analysis of Largest Phase 3 Gene Therapy Study in Adults with Severe Hemophilia A at 15th Annual Congress of European Association for Haemophilia and

SAN RAFAEL, Calif., Feb. 4, 2022 /PRNewswire/ BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that the Company presented positive r.

Debra charlesworthTraci mccartyExchange commissionBiomarin pharmaceutical incVirtual congressData monitoring committeeEuropean association for haemophiliaBiomarin pharmaceuticalAnnual virtual congressEuropean associationAllied disordersAnnualized bleeding rateValoctocogene roxaparvovec safetyMarin pharmaceutical

BioMarin Reports Hemophilia A Gene Therapy Produced Stable and Durable Annualized Bleed Control in Two Year Analysis

BioMarin Reports Hemophilia A Gene Therapy Produced Stable and Durable Annualized Bleed Control in Two Year Analysis
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United statesHank fuchsWorldwide researchCommittee for advanced therapiesEuropean medicines agencyDrug administrationCommittee for medicinal products human useUniversity of michiganMarin pharmaceuticalAnnualized bleeding rateSteven pipeMarketing authorization applicationMedicinal productsHuman useAdvanced therapiesBiologics license application

Investegate |Centessa Pharmaceuticals plc Announcements | Centessa Pharmaceuticals plc: Centessa Pharmaceuticals Announces Positive Topline Data from Proof-of-Concept Study of SerpinPC in Severe Hemophilia A and B Patients Not on Prophylaxis

Investegate |Centessa Pharmaceuticals plc Announcements | Centessa Pharmaceuticals plc: Centessa Pharmaceuticals Announces Positive Topline Data from Proof-of-Concept Study of SerpinPC in Severe Hemophilia A and B Patients Not on Prophylaxis
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United kingdomCity ofDavid lillicrapDan budwickMary clarkJennifer porcelliAntoine yverStrategic communicationsCompany nasdaqCentessa pharmaceuticals announces positive topline dataApcintex limited apcinteCentessa pharmaceuticalsWorld federation of hemophilia advisory boardMolecular medicine at queen universityProof of concept studySevere hemophilia