Family shares their Progeria journey скачать в хорошем качестве

Family shares their Progeria journey

Eiger BioPharmaceuticals, Inc. (Nasdaq:EIGR), focused on the development and commercialization of targeted therapies for serious rare and ultra-rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has approved Zokinvy™ (lonafarnib) for the treatment of Hutchinson-Gilford Progeria Syndrome (HGPS or Progeria) and processing-deficient Progeroid Laminopathies (PL). Progeria and Progeroid Laminopathies are separate and distinct ultra-rare, genetic, premature aging diseases that accelerate mortality in young patients. Disease manifestations include growth failure, loss of body fat and hair, aged-looking skin, stiffness of joints, hip dislocation, generalized atherosclerosis, cardiovascular disease and stroke. Untreated children with Progeria die of heart disease at an average age of 14.5 years. There are 20 children and young adults with Progeria and PL identified and followed in the U.S. To view the multimedia release go to: https://www.multivu.com/players/Engli...