by Peter Ciszewski | Sep 20, 2018
Greg Duncan, CEO of Celtaxsys discusses the top line results of its Phase 2 EMPIRE-CF trial evaluating oral, once daily anti-inflammatory molecule, acebilustat, for the treatment of cystic fibrosis (CF), irrespective of the causative genotype. In the 200...
by Peter Ciszewski | Sep 19, 2018
Heather A. Lau, MD, Director, Lysosomal Storage Disease Program at NYU Langone in New York City discusses Tay-Sachs disease, a rare inherited disorder that progressively destroys nerve cells (neurons) in the brain and spinal cord. Tay-Sachs disease is very rare...
by Peter Ciszewski | Sep 18, 2018
Len Walt, Vice President, Head of Medical Affairs, SOBI in North America, discusses Hemophagocytic lymphohistiocytosis (HLH), a rare condition in which the body makes too many activated immune cells (macrophages and lymphocytes). People with HLH usually develop...
by Peter Ciszewski | Sep 17, 2018
The National Institutes of Health (NIH) today announced the launch of a new initiative to help speed the development of cures for sickle cell disease, a group of inherited blood disorders affecting at least 100,000 people in the United States and 20 million worldwide....
by Peter Ciszewski | Sep 13, 2018
The US Food and Drug Administration (FDA) approved a subcutaneous formulation of tocilizumab (Actemra, Genentech) for the treatment of active systemic juvenile idiopathic arthritis in patients 2 years of age and older. “Systemic juvenile idiopathic arthritis is a...
