FDA Approves New Treatment for Hemophilia Patients

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)....

Farber Disease Natural History Study

  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...

Hereditary Angioedema (HAE)

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...

Fulcrum Therapeutics

  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)...