A study led by BRC-supported researcher Marie Scully has been chosen as one of the most outstanding manuscripts published in Blood.
Dr Scully is a consultant haematologist at UCLH and her publication focuses on the use of a recombinant enzyme (ADAMTS-13) to treat the rare blood clotting disorder - Thrombotic thrombocytopenic purpura (TTP). The work carried out was a first-in-human phase 1 study, showing that the recombinant enzyme ADAMTS-13 was safe, did not trigger an immune response and was tolerated in TTP.
This year over 1,000 manuscripts were published in Blood, the Journal of the American Society of Haematology. Editors of the journal selected the top ten manuscripts based on quality of the work and interest within the field of haematology.
UCLH is the biggest international specialist centre for TTP and Marie who leads the TTP research group. TTP is caused by the lack of ADMATS-13, resulting in blood clots to form in small blood vessels throughout the body. These clots can limit or block the flow of oxygen-rich blood to organs leading to health problems such as strokes or epileptic events, heart or kidney damage.
To read Marie’s chosen manuscript, please see Blood.
