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UK's MHRA Revokes Authorisation for Novartis' Sickle Cell Drug

Britain's health regulator said on Wednesday it had revoked a conditional marketing authorisation for Novartis' sickle cell disease drug, Adakveo. ....

Switzerland , Bengaluru , Karnataka , India , United-kingdom , Swiss , Britain , Eva-mathews , Shinjini-ganguli , European-medicines-agency , Regulatory-agency , Reuters

Sickle Cell CRISPR Therapy May Offer 'Functional Cure'

Patients with severe sickle cell disease could soon be freed from vaso-occlusive crises after receiving an infusion of exa-cel, the first gene-editing therapy to use CRISPR technology. ....

United-states , Memphis , Tennessee , Ohio , California , Columbus , American , Sarahh-obrien , Haydar-frangoul , Akshay-sharma , Vertex-pharmaceuticals , American-society-of-hematology

Bluebird Signs Pact With Insurer for Sickle Cell Gene Therapy

Bluebird bio said on Thursday a large insurer had agreed to cover its newly approved sickle cell disease gene therapy, easing some investor worries about resistance from. ....

Bengaluru , Karnataka , India , United-states , Krishna-chandra-eluri , David-nierengarten , Sriparna-roy , Leroy-leo , Jack-allen , Vertex-pharmaceuticals , Reuters , Genomics-genomic-medicine

SCD Mortality Rates Improved for Black Patients in 2010s

The average age of death for Black US patients with sickle cell disease rose from 39 years in 1999-2009 to 43 years in 2010-2020, reflecting improvements in treatment. ....

United-states , American , Kristinea-karkoska , Medscape-professional-network , American-society-of-hematology , National-heart , University-of-cincinnati-college-medicine , Blood-institute , Cincinnati-college , American-society , Medscape-professional

Europe Approves Avapritinib for Rare Mast Cell Disease

The drug targets a mast cell mutation that gives rise to indolent systemic mastocytosis, a rare and debilitating blood disorder. ....

Germany , European-union , European-commission , Medicinal-products , Human-use , Rare-diseases , Europe , Uropean , Lysosomal-storage-disease , Systemic-mastocytosis , Blood