Malignant bone marrow disease: New hope for MPN patients: Myeloproliferative neoplasms (MPNs) — a group of rare but malignant bone marrow disorders

MPNs are a group of rare, malignant diseases of the bone marrow involving the production of an excess of red blood cells, white blood cells and/or platelets. MPNs are chronic diseases with only 1 to 2 new cases diagnosed per 100,000 people every year. MPNs can affect people at any age, but they are most common among adults around 60 […]

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Early clinical trial data show gene therapy reversing sickle cell anemia: Treatment uses reduced-intensity preconditioning to enhance its global transportability

After over a decade of preclinical research and development, a new gene therapy treatment for sickle cell anemia (SCA) is reversing disease symptoms in two adults and showing early potential for transportability to resource-challenged parts of the world where SCA is most common. Preliminary data from a pilot Phase 1-2 clinical trial testing the gene-addition therapy were presented Dec. 3 […]

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New Hope for African Children With Sickle-Cell Disease

A drug that protects children in wealthy countries against painful and sometimes lethal bouts of sickle-cell disease has been proven safe for use in Africa, where the condition is far more common, scientists reported on Saturday. More research remains to be done, experts said, but knowing that hydroxyurea — a cheap, effective and easy-to-take pill — can safely be given […]

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How your muscles form

All vertebrates need muscles to function; they are the most abundant tissue in the human body and are integral to movement. In a recent article published in Nature Communications, an international team of researchers discovered two proteins essential to the development of skeletal muscle. This research, led by Jean-François Côté, a professor at the Montreal Clinical Research Institute (IRCM) and […]

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