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.
Source: medscape.com
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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.
Source: medscape.com
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...
Source: medscape.com
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.
Source: medscape.com
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.
Source: medscape.com