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Clinical trial successfully repurposes cancer drug for hereditary bleeding disorder!
Here is a link to the publication of the research:
http://dx.doi.org/10.1056/NEJMoa2312749
“Hereditary hemorrhagic telangiectasia (HHT), the world's second-most-common inherited bleeding disorder, affects approximately 1-in-5,000 people and can have life-threatening complications, but there are currently no U.S. FDA-approved drugs to treat HHT.”
“The results of our trial demonstrate the clear safety and efficacy of pomalidomide to treat bleeding in HHT, giving these patients a much-needed effective treatment option," said first author Hanny Al-Samkari, MD, the Peggy S. Blitz Endowed Chair in Hematology/Oncology at Massachusetts General Hospital.
#inspiration
Here is a link to the publication of the research:
http://dx.doi.org/10.1056/NEJMoa2312749
“Hereditary hemorrhagic telangiectasia (HHT), the world's second-most-common inherited bleeding disorder, affects approximately 1-in-5,000 people and can have life-threatening complications, but there are currently no U.S. FDA-approved drugs to treat HHT.”
“The results of our trial demonstrate the clear safety and efficacy of pomalidomide to treat bleeding in HHT, giving these patients a much-needed effective treatment option," said first author Hanny Al-Samkari, MD, the Peggy S. Blitz Endowed Chair in Hematology/Oncology at Massachusetts General Hospital.
#inspiration
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