Children’s Hospital of The King’s Daughter’s Center for Novel Therapeutics and Gene Therapy was created to combine clinical research and clinical care. As Director of Neurology, Crystal Proud, MD, explains how diagnosing rare diseases and incorporating a standard of care has extended into revolutionary research and help for families all over the United States.
I'm Crystal Proud. I am a neuromuscular neurologist and the Director of Neurology at the Children's Hospital of the King's Daughters in Norfolk, Virginia. The Center for Novel Therapeutics and Gene Therapy at CHKD is a center that was created in order to try and combine clinical research and clinical care because right now, there is this evolution uh that goes from diagnostics of rare disease to incorporating standard of care and then extending into research that's really revolutionary and then making a full circle. Once that treatment gets FDA approved into continuing that clinical care, it's been really exciting to be able to bring very competitive research to CHKD. And we've really been able to draw families from truly all over the United States. We've had global interest as well. So we do our gene transfer therapies here on the Neuroscience Unit at CHKD and our nurses. There are very familiar with our protocols. Our vascular access team is very familiar with placing our IVS and the floor. There is very familiar with having our patients up there for gene transfer therapy and the excitement that that brings to, we really rely on collaborating with our pediatricians because we need to be able to have a medical home for our kids. Um We need someone to provide that continuity when they're sick, but also to be able to reach out to us when there may be a different consideration that needs to be made because they have a neuromuscular disease. You know, when we think about things like duchin muscular, dystrophy, spinal muscular atrophy and some other neuromuscular conditions. It's not uncommon for um people to not be aware that we have effective life saving treatments. Um And in fact, the the community that may not be as aware that there are these opportunities are the specialists that provide care to pregnant women. Long term positive outcomes are not something that I think are yet widely dispersed. But we really need to make all specialists aware whether they're taking care of a mom, a baby, a growing child, that there is an incredible amount of hope and optimism. There are therapies, these Children are living longer healthier lives. And so we want to provide encouragement to the families. Gene therapy is absolutely incredible and revolutionary in providing such a new hope for our patients with neuromuscular disease. However, it is not something to be taken lightly. It is imperative that Children receive this therapy at a center of excellence that has experience and expertise in administering gene transfer therapy. It does not come without the potential for side effects. And fortunately, here at our novel therapeutics and gene therapy Center at CHKD. We are very familiar with navigating potential adverse effects, optimizing outcomes and providing comprehensive support for patients that get gene therapy. So it's absolutely key that patients receive their care in an experienced um location that has this comprehensive multidisciplinary team.
