A single one-time gene therapy could free patients with α-thalassemia, a rare and debilitating blood disorder, from the burden of lifelong transfusions. A single one-time gene therapy could free ...
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One match, one chance: Welsh mom fights rare blood cancer
A single mother in Wales is sharing her story after learning that just one person in the world may hold the key to saving her ...
WASHINGTON — The Food and Drug Administration has approved the first drug treatment for pediatric patients 12 and older with a rare blood disorder. The treatment for acquired thrombotic ...
Add Yahoo as a preferred source to see more of our stories on Google. A new "proteomic" blood test can accurately screen a newborn's DNA for thousands of rare, inherited diseases by analyzing just a ...
DAVANAGERE: Even though he himself is a haemophilia patient, it did not deter him from becoming a champion to protect the ...
CAMBRIDGE, Mass., May 14, 2025 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (AGIO), a leader in cellular metabolism and pyruvate kinase (PK) activation pioneering therapies for rare diseases, today ...
A single, untargeted proteomics test for rare genetic diseases has been developed. A research team from the University of Melbourne (Australia) and Murdoch Children’s Research Institute (Victoria, ...
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