Tag: <span>rare blood disease</span>

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Predicting which patients with a rare blood disease will respond to the only FDA-approved treatment
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Predicting which patients with a rare blood disease will respond to the only FDA-approved treatment

by  Perelman School of Medicine at the University of Pennsylvania Credit: CC0 Public Domain New research has uncovered a precision medicine test using blood proteins to identify a novel patient subgroup of idiopathic multicentric Castleman disease (iMCD), a rare blood disorder, who are more likely to respond to siltuximab, the only FDA approved treatment for the...

Discovery could lead to new treatment for rare blood disease
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Discovery could lead to new treatment for rare blood disease

University of Alberta cancer researcher proposes treating cutaneous T-cell lymphoma before it reaches the skin UNIVERSITY OF ALBERTA FACULTY OF MEDICINE & DENTISTRY A cancer researcher at the University of Alberta has made a discovery that could unlock new ways to treat a rare blood disease. By taking biopsies of skin lesions from patients suffering...

Six patients with rare blood disease are doing well after gene therapy clinical trial
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Six patients with rare blood disease are doing well after gene therapy clinical trial

by Sarah C.p. Williams, University of California, Los Angeles UCLA researchers are part of an international team that reported the use of a stem cell gene therapy to treat nine people with the rare, inherited blood disease known as X-linked chronic granulomatous disease, or X-CGD. Six of those patients are now in remission and have...