by Peter Ciszewski | Jul 31, 2019
John Maslowski is President and Chief Executive Officer (CEO) of Fibrocell Science, a biotech company that is developing cell and gene therapies for a variety of rare, mostly skin-related, conditions. At the BIO International Convention held in Philadelphia,...
by Peter Ciszewski | Jul 30, 2019
Nick Kenny, PhD, Chief Scientific Officer at Syneos Health, understands the important role patients play in developing orphan drugs and designing clinical trials. Recently, we talked to Dr. Kenny at the BIO International Convention held in Philadelphia, PA. Dr....
by Peter Ciszewski | Jul 29, 2019
Ido Weinberg, MD, Medical Director, VASCORE; Assistant Professor of Medicine at Harvard Medical School, Co-Medical Director, Anticoagulation Management Services, Massachusetts General Hospital, discusses diagnostic challenges of treatment limitations of...
by Peter Ciszewski | Jul 24, 2019
The FDA granted Orphan Drug Designation to a novel menin-mixed lineage leukemia (menin-MLL) inhibitor KO-539 for the treatment of acute myeloid leukemia (AML). KO-539 is a selective small molecule inhibitor of the menin-MLL protein-protein interaction. MLL-rearranged...
by Peter Ciszewski | Jul 24, 2019
KAT6A syndrome is a rare genetic condition as a result of a mutation in the KAT6A gene. That gene produces the KAT6A protein that is instrumental in DNA packaging and the production of other proteins in the cell. It is an extremely rare condition with only 195...
