Retinoid Therapy for Congenital Ichthyosis Shows Promise

  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...

Managing Farber Disease

  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...

Global Genes CEO Discusses Partnership with Rare-X

  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...