by Peter Ciszewski | Apr 2, 2020
Geoff MacKay, president and CEO of Avrobio, described the company’s history and philosophy for developing ex vivo gene therapies for various diseases, such as Pompe disease, Fabry disease, Gaucher disease, and cystinosis. As Mackay explains in this video, the...
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...
