by CheckRare Staff | Feb 13, 2024
Jörg Schüttrumpf, MD, Chief Scientific Innovation Officer at Grifols, discusses the Phase 1/2 study evaluating a breakthrough treatment option for alpha1-antitrypsin deficiency. Alpha-1 antitrypsin deficiency (AATD) is an inherited disease caused by...
by CheckRare Staff | Feb 10, 2024
Jatin Shah, MD, PhD, of Sumitomo Pharma, discusses two treatments in Phase 1 trials for myelofibrosis and acute myeloid leukemia. Jatin Shah, MD, PhD, of Sumitomo Pharma, discusses two treatments in Phase 1 trials for myelofibrosis and acute myeloid...
by CheckRare Staff | Feb 9, 2024
Steven Pipe, MD, of the University of Michigan, discusses the results of HAVEN 7, a Phase 3 trial testing the efficacy and safety of emicizumab to prevent bleeding episodes in patients under 12 months of age with hemophilia A. Hemophilia A is a...
by CheckRare Staff | Feb 7, 2024
Giacomo Chiesi, Head of Chiesi Global Rare Diseases and co-author of the report “Rare Disease Burden of Care and the Economic Impact of Citizens in Germany, France, and Italy”, discusses report in detail. There are an estimated 35-36 million...
by CheckRare Staff | Feb 6, 2024
Kim Smith-Whitley, MD, chief medical officer for Real-World Evidence, Patient Advocacy, and External Collaborations at Pfizer and pediatric hematologist at the Children’s Hospital of Philadelphia, discusses recent advances in sickle cell disease treatment. ...
