Bar Harbor, Maine – In the battle against cancer, allies can come from unexpected sources. Research at The Jackson Laboratory has yielded a new approach to treating leukemia, one that targets leukemia-proliferating cells with drugs that are already on the market. Jackson Adjunct Professor Shaoguang Li, M.D., Ph.D., who now...
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BURTON, Ohio — The first signs that something was wrong with 11-year-old Connor Teare came when he was a toddler. His muscles were growing increasingly rigid and becoming more difficult to move. He went from leg braces to a walker and, finally, a wheelchair by the time he was 5....
A new policy framework is the only way to ensure that progress made for the 30 million people living with a rare disease in Europe is not left to luck or chance during the next decade. The eight Rare 2030 recommendations, the result of the two-year participative Foresight Study led by EURORDIS-Rare...
A drug derived from the hydrangea root, used for centuries in traditional Chinese medicine, shows promise in treating autoimmune disorders, report researchers from the Program in Cellular and Molecular Medicine and the Immune Disease Institute at Children’s Hospital Boston (PCMM/IDI), along with the Harvard School of Dental Medicine. In the...
In recent years, tumor immunotherapy has emerged as a highly promising and much-touted oncological approach. It is based on using humanized antibodies called immune checkpoint inhibitors (ICIs) to block the cellular pathways that inhibit the activity of T lymphocytes, a type of immune system cells that help protect the body...
PHILADELPHIA-The study of a disease is inherently challenging when patients are few and far between, but doctors at the Perelman School of Medicine at the University of Pennsylvania have reported a new “patient-powered” approach that may help to revolutionize the study of rare diseases. The Penn Medicine researchers, in a...
College Isn’t Easy, Especially during a pandemic College isn’t easy. Trust me, I’ve tried, and it was not for me. I think highly of anyone that makes it through college but with honors, I am doubly impressed. That’s just what Rosie Correll did when she graduated with honors and on...
A vote for Darius could mean a $100,000 donation in the fight against Muscular Dystrophy! Earlier this month, we brought you the story of Darius Weems and his quest to raise money and awareness to Duchenne Muscular Dystrophy- the number one genetic killer of children. Darius has DMD. It’s the...
CAMBRIDGE, Mass. – AavantiBio, a gene therapy company focused on transforming the lives of patients with rare genetic diseases, today announced the appointment of Christopher Wright, M.D., Ph.D., as Chief Medical Officer. A neurologist and neuroscientist, Dr. Wright brings to AavantiBio more than 20 years of medical research and drug...
CAMBRIDGE, Mass. – AavantiBio, a gene therapy company focused on transforming the lives of patients with rare genetic diseases, today announced the appointment of Ty Howton as Chief Operating Officer and General Counsel. A veteran life sciences executive, Mr. Howton brings to AavantiBio more than two decades of strategic, operational,...