by Peter Ciszewski | Dec 8, 2020
Ron Cooper, President and CEO of Albireo Pharma discusses the results from two clinical trials assessing odevixibat to treat progressive familial intrahepatic cholestasis (PFIC). PFIC is a rare genetic disorder that causes progressive, life-threatening liver...
by Peter Ciszewski | Dec 7, 2020
Kristin Highland, MD of the Cleveland Clinic discusses the results for the ongoing SENSCIS-ON trial testing nintedanib to treat interstitial lung disease in patients with systemic sclerosis. Systemic sclerosis is a rare autoimmune disease that is associated...
by Peter Ciszewski | Dec 4, 2020
The U.S. Food and Drug Administration (FDA) has approved Orladeyo (berotralstat), a once-daily, pill as a prophylaxis treatment for hereditary angioedema (HAE) attacks. More specifically, the drug is indicated to treat adults and pediatric patients 12 years and older...
by Peter Ciszewski | Dec 4, 2020
Fariba Navid, MD, Associate Professor of Clinical Pediatrics, Keck School of Medicine of USC, announces her involvement in a trial for nirogacestat as a treatment for Desmoid tumors in pediatric patients. Desmoid tumors are benign tumors that can grow anywhere...
by Peter Ciszewski | Dec 3, 2020
Irina Anselm, MD, Director of the Mitochondrial Program and Co-Director of the Neurometabolic Program at Boston Children’s Hospital explains why aromatic L-amino acid decarboxylase (AADC) deficiency is often misdiagnosed for more common conditions, such...
