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Vertex Announces Approval of First CRISPR/Cas9 Gene-Edited Therapy, CASGEVY™, for the Treatment of Sickle Cell Disease (SCD) and Transfusion-Dependent Beta Thalassemia (TDT) in Kingdom of Saudi Arabia

- CASGEVY™ is first medicine ever to be evaluated through the SFDA’s Breakthrough Medicines Program - - First treatment center in Saudi Arabia has been activated - .

United statesCity ofUnited kingdomSaudi arabiaKingdom of saudi arabiaReshma kewalramaniDrug authorityCompanies to workTreatment centerVertex pharmaceuticals incorporated nasdaqMinistry of national guard health affairsExchange commissionVertex pharmaceuticals incorporatedSaudi foodMarketing authorizationChief executive officer

PTC Therapeutics (PTCT) Provides an Update on Commercial Progress and R&D Pipeline

PTC Therapeutics (PTCT) Provides an Update on Commercial Progress and R&D Pipeline
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Matthewb kleinCommittee for medicinal products humanPtc therapeutics incBlackstone life sciencesEuropean commissionTherapeutics incEuropean medicines agencyDrug administrationHealthcare conferenceChief executive officerRoyalty pharmaBlackstone lifeKey clinicalRegulatory eventsFull yearPayments full year

PTC Therapeutics Provides an Update on Commercial Progress and R&D Pipeline at 42nd Annual J P Morgan Healthcare Conference

– Unaudited 2023 total revenue of $946 million, representing 35% year-over-year growth – – Regulatory filings in the EU and US for sepiapterin in PKU, a potential $1 billion global commercial.

United statesKylie okeefeMatthewb kleinJeanine clementeEuropean medicines agencyTherapeutics incBlackstone life sciencesCommittee for medicinal products humanEuropean commissionDrug administrationHealthcare conferenceChief executive officerRoyalty pharmaBlackstone lifeKey clinicalRegulatory events

PTC Therapeutics Provides an Update on Commercial Progress and R&D Pipeline at 42nd Annual J P Morgan Healthcare Conference

PTC Therapeutics Provides an Update on Commercial Progress and R&D Pipeline at 42nd Annual J P Morgan Healthcare Conference
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United statesKylie okeefeMatthewb kleinJeanine clementeCommittee for medicinal products humanEuropean commissionDrug administrationEuropean medicines agencyTherapeutics incBlackstone life sciencesHealthcare conferenceChief executive officerRoyalty pharmaBlackstone lifeKey clinicalRegulatory events

Research and Markets: MONJUVI (tafasitamab-cxix) Drug Insights and Market Forecasts, 2019-2022 and 2023-2032: Focus on 7MM - United States, Germany, France, Italy, Spain, United Kingdom, and Japan

Research and Markets: MONJUVI (tafasitamab-cxix) Drug Insights and Market Forecasts, 2019-2022 and 2023-2032: Focus on 7MM - United States, Germany, France, Italy, Spain, United Kingdom, and Japan
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United kingdomUnited statesStefan klotterLaura woodE st office hours callEuropean commissionClinical developmentHealth canadaOffice hours callMarket forecastDiffuse largeb cellMarketing authorizationTopics coveredDevelopmental activitiesMarketed therapiesLate stage emerging therapies