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– Data from 75 patients with transfusion-dependent beta thalassemia or severe sickle cell disease with follow-up of up to 37.2 months continue to demonstrate that exa-cel has the potential to be a one-time functional cure –– Safety profile generally consistent with myeloablative conditioning and autologous stem cell...
- Vertex announces three additional abstracts on the burden of beta thalassemia and sickle cell disease accepted for poster presentation - Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR
- Vertex announces three additional abstracts on the burden of beta thalassemia and sickle cell disease accepted for poster presentation –BOSTON & ZUG, Switzerland & CAMBRIDGE, Mass.--(BUSINESS WIRE)--Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR Therapeutics (NASDAQ: CRSP) today announced new late-...
Background: Real-world effectiveness of interventions in palliative care need to be systematically quantified to inform patient/clinical decisions. Neuropathic pain is prevalent and difficult to palliate. Tricyclic antidepressants have an established role for some neuropathic pain aetiologies, but this is less clear in palliative care. Aim: To describe the real-world use and outcomes from amitriptyline or nortriptyline for neuropathic pain in palliative care. Design: An international, prospective, consecutive cohort post-marketing/phase IV/pharmacovigilance/quality improvement study of palliat...
Background: People with life-limiting illnesses experience a range of distressing symptoms. Appetite-related symptoms are common, but studies have found varied prevalence and the distress caused has had limited quantification. Objectives: To examine the clinicodemographic factors and trajectory of appetite-related distress in the last 60 days of life. Design/Setting/Subjects: Consecutive cohort of 109,385 patients (359,038 data points) using specialist palliative care services in the Australian Palliative Care Outcomes Collaboration (PCOC). Measurements: Patient-reported appetite-related distr...