Recent publications in JAMA Network Open, Expert Evaluation of Strategies to Modernize Newborn Screening in the United States call for urgent change to ensure the U.S. newborn scre...

In the first 24 to 48 hours of a baby’s life, a small blood sample is taken to detect serious genetic conditions, some of them deadly, that can be treated if diagnosed early.1 Than...

Gene therapy is designed to insert functional copies of a faulty gene that is not working properly. Genes are the genetic blueprints for proteins and sometimes a mutation can resul...

The gene therapy field is on the cusp of something special in realizing the transformative potential of gene therapies for patients and their families. After a year as president an...

As we pass the one-year mark of the beginning of the COVID-19 pandemic, the collective shift to at-home work, and my transition to the role of chief executive officer at Orchard, I...

This article was originally published as a Guest Column on Cell & Gene. The transformative potential of gene therapy is bringing renewed hope to the rare disease community, but...