by Peter Ciszewski | Oct 10, 2021
Deborah Marsden, MD, Global Medical Expert, Medical Affairs, Ultragenyx, discusses the diagnostic journey for many patients with mucopolysaccharidosis type VII (MPS VII). MPS VII is a rare lysosomal storage disorder. The severity of MPS VII varies widely among...
by Peter Ciszewski | Oct 10, 2021
Rudolf Baumgartner, MD, Chief Medical Officer and Head of Clinical Development at Saniona, gives an overview of Prader-Willi syndrome (PWS) and tesomet, a drug combination under investigation for the treatment of PWS. As Dr. Baumgartner explains, PWS is a rare...
by Peter Ciszewski | Oct 8, 2021
The Food and Drug Administration (FDA) has approved avacopan (Tavneos) as an adjunctive treatment for adult patients with severe active anti-neutrophil cytoplasmic autoantibody (ANCA)-associated vasculitis. ANCA-associated vasculitis is a systemic disease in which...
by Peter Ciszewski | Oct 8, 2021
Ron Cooper, President and CEO of Albireo Pharma gives an overview of progressive familial intrahepatic cholestasis (PFIC). As Mr. Cooper explains, PFIC is a rare genetic disorder that causes progressive, life-threatening liver disease due to an inability to...
by Peter Ciszewski | Oct 8, 2021
Jennifer Gudeman, PharmD, Vice President of Medical and Clinical Affairs at Avadel Pharmaceuticals, describes how narcolepsy is diagnosed and why it often takes so long to get a proper diagnosis. Narcolepsy is a rare neurological disorder characterized by...
