by Peter Ciszewski | Nov 29, 2021
Anthony Quinn, PhD, President and CEO of Aeglea BioTherapeutics, discusses the PEACE study, a phase 3 clinical trial of pegzilarginase for the treatment of arginase 1 (ARG1) deficiency. ARG1 deficiency is a rare genetic disorder characterized by complete or...
by Peter Ciszewski | Nov 26, 2021
Alan Mendelsohn, MD, Chief Medical Officer at Timber Pharmaceuticals, gives an update on their congenital ichthyosis clinical trial. Congenital ichthyosis is a rare genetic skin disorder characterized by dry, thickened, and scaling skin. Individuals with this...
by Peter Ciszewski | Nov 24, 2021
Dinesh Khanna, MD, Professor of Rheumatology and Medicine at the University of Michigan, discusses Horizon Therapeutics’ phase 2b clinical trial of HZN-825, to treat patients with diffuse cutaneous systemic sclerosis (dcSSc). dcSSc is a subtype of systemic...
by Peter Ciszewski | Nov 23, 2021
Alexander Solyom, MD of Aceragen provides an overview of the treatment options for Farber disease. Farber disease is a lysosomal storage disorder due to a deficiency of acid ceramidase that leads to the accumulation of ceramide. The most common symptoms of...
by Peter Ciszewski | Nov 22, 2021
Craig Martin, CEO of Global Genes, discusses the organization’s recent partnership with Rare-X, a technology non-profit that securely gathers, structures, and shares patient-owned data. As Mr. Martin explains, Rare-X is an organization that gathers rare...
