April 18th 2024
A cost-utility analysis conducted from the perspective of the Italian health system found that pegcetacoplan was more effective and less costly than 2 complement 5 (C5) inhibitors for the treatment of paroxysmal nocturnal hemoglobinuria (PNH).
What We’re Reading: Pharmacy Staff Walkout; Male Parental Leave; FDA Reviewing SCD Therapy
October 30th 2023Pharmacy workers embark on a multiday strike; Fathers are taking paid family leave to bond with their newborns; the FDA is set to evaluate a pioneering gene therapy for sickle cell disease (SCD).
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Relative Benefit in Survival, Reduction in TE/MAVE Risk With Eculizumab for Patients With PNH
September 19th 2023When patients with paroxysmal nocturnal hemoglobinuria (PNH) received eculizumab, survival rates increased and thromboembolic events/major adverse vascular events (TE/MAVE) decreased.
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Study Finds Simulation Useful to Boost Awareness of Osteomyelitis in SCD Among Residents, Fellows
August 28th 2023A simulation case for emergency medicine residents was found to be helpful as a review of the medical diagnosis and management of osteomyelitis in patients with pediatric sickle cell disease (SCD).
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Study Validates First Disease-Specific PRO Instrument for Patients With Congenital TTP
July 27th 2023Congenital thrombotic thrombocytopenic purpura (TTP), an ultra-rare disease, can significantly impact quality of life, but clinicians have previously lacked a disease-specific way to track experiences via patient-reported outcomes (PROs).
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