News

Cash-strapped governments across the 28-member European Union are struggling to control runaway healthcare expenditures — at exactly the same time as the promise of new but expensive therapies to treat rare diseases has never been greater. That’s the paradox faced by pharmaceutical companies as well as patient advocacy groups in…

Bluebird bio presented new positive results from the ongoing Phase 1/2 HGB-206 clinical trial evaluating the effectiveness and safety of investigational gene therapy LentiGlobin in patients with severe sickle cell anemia. The company had previously revealed findings from the trial at the end of last year and earlier this…

Global Blood Therapeutics’ lead therapeutic candidate voxelotor can induce rapid, robust, and sustained improvements in the amount of hemoglobin in adolescents and adults with sickle cell disease (SCD), according to interim results from the first part of a Phase 3 trial. The latest results of GBT’s clinical…

Hemolytic transfusion reactions, a serious complication of red blood cell transfusion, can be difficult to diagnose in patients with sickle cell disease and even be misinterpreted as severe vaso-occlusive crisis. A report published in the journal Transfusion highlights the importance of accurate and prompt diagnosis of such complications in…

For this year’s Solutions to Empower Patients (STEP) program, global healthcare company Novartis has announced it will fund up to five original and creative ideas to help sickle cell disease (SCD) patients and their families. Each selected proposal will be awarded up to $50,000. Specifically, Novartis is seeking…

A new clinical decision support-based tool can help primary care physicians improve the care and management of patients with sickle cell disease (SCD), a study reports. The study, “Effectiveness of Clinical Decision Support Based Intervention in the Improvement of Care for Adult Sickle Cell Disease Patients in…

Students with sickle cell disease (SCD) feel that educators and administrators at their schools lack knowledge about SCD management — an issue that could be addressed by developing a handbook that staff could use to help students with SCD. The study with that finding, “Student perspectives on managing sickle…

Modus Therapeutics is going to launch a Phase 1 clinical study in the United States to evaluate the potential of its lead candidate sevuparin for the treatment of sickle cell disease (SCD). In the trial, the company will broaden the clinical scope and usefulness of sevuparin to treat…

A virtual reality program designed to provide a distraction from pain may be an effective complementary therapy for children who are hospitalized for a sickle cell crisis, a study shows. The study, “Virtual reality as complementary pain therapy in hospitalized patients with sickle cell disease,” was published in the journal…

With the U.S. midterm elections now less than two weeks away, patient advocacy groups are solidly focused on a range of hot-button issues, from the Orphan Drug Tax Creditand affordable health insurance to future funding for rare disease research. Yet “whether Democrats take over the House or Senate, or…