by Peter Ciszewski | Apr 1, 2020
The U.S. Food and Drug Administration (FDA) has approved Sevenfact [coagulation factor VIIa (recombinant)-jncw] to treat and control of bleeding episodes that occur in persons 12 years of age and older with hemophilia A or B with inhibitors (neutralizing antibodies)....
by Peter Ciszewski | Apr 1, 2020
Epidemiology of Covid-19 in a Long-Term Care Facility in King County, Washington Dr. McMichael and colleagues provided a detailed assessment of the persons who became infected with Covid-19 in a long-term care facility in Seattle, Washington. As of March 18, there...
by Peter Ciszewski | Mar 31, 2020
John Mitchell, MD, pediatric endocrinologist at Montreal Children’s Hospital and McGill University, explains the need for a natural history study involving Farber disease. Farber disease is a lysosomal disorder due to mutations of the ASAH1 gene that leads to...
by Peter Ciszewski | Mar 30, 2020
Hereditary angioedema (HAE) is a rare genetic disease that has gone through a revolution in the past decade with numerous medications now available to treat people with HAE. Unfortunately, identifying those people remains a challenge for those unfamiliar with the...
by Peter Ciszewski | Mar 27, 2020
Diego Cadavid, MD, of Fulcrum Therapeutics explains the philosophy of the company and how they are developing therapies to stop certain mutated genes from overproducing certain proteins. One such therapy is losmapimod, a mitogen-activated protein kinase (MAPK)...