by Peter Ciszewski | Apr 24, 2020
Recently, we spoke to Beverley Francis-Gibson, President of the Sickle Cell Disease Association of America (SCDAA) about what her organization is doing to assist patients with sickle cell disease during the current COVID-19 pandemic. SCDAA is a non-profit organization...
by Peter Ciszewski | Apr 23, 2020
Dwight Koeberl, MD, PhD of Duke University School of Medicine explains the difference between enzyme replacement therapy (ERT) and gene therapy, as it relates to lysosomal storage disorders like Pompe disease. Dr. Koebel is currently conducting a gene therapy...
by Peter Ciszewski | Apr 22, 2020
Riam Shammaa, MD, Founder and CEO of INTELLiSTEM Technologies, talks about some of the hurdles his company has to overcome to develop a vaccine for COVID-19, during the lockdown. As Dr. Shammaa explains in this video, one of the more surprising problem they had...
by Peter Ciszewski | Apr 21, 2020
The U.S. Food and Drug Administration (FDA) has approved the kinase inhibitor, Pemazyre (pemigatinib), to treat adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or...
by Peter Ciszewski | Apr 20, 2020
Shannon Palmatier is the mother of two boys with progressive familial intrahepatic cholestasis (PFIC), a rare hereditary disease in which children are unable to properly remove bile acids from their liver. The disease is an autosomal recessive disorder and is...
