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The manner in which technological advances have changed the way we live is nothing short of amazing. So many free applications exist to help us with every aspect of life, from shopping to studying, and from cooking to entertainment. If we can use apps for all of this and more, why don’t we use them to help manage our health? Here are some of the apps I use that have been extremely useful: Waterlogged. It...


LentiGlobin, Bluebird Bio’s investigational gene therapy for sickle cell disease (SCD), continues to show promising results in SCD patients participating in the company’s Phase 1/2 HGB-206 clinical trial, according to the latest study data. The new findings — which included data from additional patients treated in the trial, updated data from those previously reported, and exploratory analyses — were presented at the 61st American Society of Hematology (ASH) Annual Meeting and Exposition, held Dec. 6-10...


With so much recent publicity surrounding gene therapy, it’s no surprise that the topic was a major focus of the recent 2019 NORD Rare Diseases & Orphan Products Breakthrough Summit. From diagnosis and clinical trial design to manufacturing, pricing strategies, and ethical concerns, gene therapy — both its high costs and limitless potential for curing diseases — generated much discussion at the Washington, D.C., event, which was organized by the National Organization for Rare Disorders...


A Phase 3 trial is testing a higher dose of Adakveo (crizanlizumab), the first approved targeted treatment for sickle cell disease, to explore if that dose might be more effective — while still safe — in easing the painful vaso-occlusive crises (VOCs) patients can have each year, according to its developer, Novartis. It is one of five ongoing studies looking at Adakveo in a variety of people with sickle cell disease (SCD), part of Novartis’ SENTRY...


When we have a crisis or another sickle cell-related complication, we often require urgent medical attention and hospital admission. So we make our way to our local hospital’s accident and emergency department or emergency room. The ER is known for the long wait to be seen by a medical professional. When I go to the ER, I hear people complain about the two to three hours’ wait, but the delay is often longer. I once...


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