Lysosomal storage diseases are progressive, inherited disorders of metabolism, many with devastating effects on the nervous system. Some of these diseases, like Infantile-Onset Pompe disease, can be treated to some degree in childhood by giving back the missing enzyme - a strategy called enzyme replacement therapy, or ERT. But a recent study reported in the New England Journal of Medicine goes a step farther -- by giving enzyme replacement therapy to a fetus with Pompe disease, for the first time, in an attempt to treat the disease before it does any serious damage. The paper’s first author is Dr. Jennifer Cohen, a Pediatric Medical Genetics Physician-Scientist at Duke University Health System. During this episode, she’ll walk us through the potential benefits of in-utero enzyme replacement therapy and other cutting-edge approaches to treating genetic neurologic conditions. A relatively new field called fetal therapy. Dr. Cohen was interviewed by Dr. Adeline Goss, the host of ANA Investigates. Series 4, Episode 10

Featuring:

• Guest: Dr. Jennifer Cohen, Duke University Health System
• Interviewer: Dr. Adeline Goss, Highland Hospital
• Disclosures: Dr. Cohen is a consultant with Bayer.