22q11.2 Deletion Syndrome (also known as 22q)

Melissa P. Wasserstein, MD, Chief of Pediatric Genetic Medicine at the Children’s Hospital at Montefiore, New York City discusses 22q11.2 deletion syndrome, a disorder caused by the deletion of a small piece of chromosome 22. The deletion occurs near the middle...

Myths Surrounding the Orphan Drug Act

Douglas Paul, PharmD, PhD discusses some of the myths surrounding the Orphan Drug Act, especially that Pharma and Biotech companies are gaming the system. These myths, Dr. Paul believes, could have long-term negative effects on Rare Disease drug development.