Insuring patient access and affordability for treatments for rare and ultra-rare diseases

(American College of Medical Genetics and Genomics) The last decade has seen tremendous progress in the development of new drugs for patients with genetic disorders, including Cystic Fibrosis, many lysosomal storage disorders (Gaucher disease, Fabry disease and others) and most recently, Duchenne Muscular Dystrophy and Spinal Muscular Atrophy (SMA). The American College of Medical Genetics and Genomics (ACMG) is concerned with the staggering projected cost of these new treatments.

from EurekAlert! - Social and Behavioral Science https://ift.tt/2Qda8EN