The Impact of Gene Therapy with Dave Novak & Rochelle Ginter

Today on Beyond the Bleeds, we welcome Rochelle Ginter, RN, at the Hemophilia Outreach Center in Green Bay, Wisconsin, and David Novak, who shares his experience receiving gene therapy for hemophilia A. We delve into the mechanics of hemophilia A, the latest advancements in gene therapy, and David’s journey in transforming his treatment approach. The conversation highlights expectations, impacts, and the broader implications of gene therapy in managing hemophilia.

Rochelle Ginter is a registered nurse with specialized hemophilia care expertise at the Hemophilia Outreach Center. Her deep understanding of hemophilia treatments and patient support has made her invaluable in advancing care and educational outreach. David Novak, a recipient of the groundbreaking gene therapy Roctavian, shares his journey navigating severe hemophilia A from birth to benefiting from modern gene therapy, providing a personal look at its transformative potential.

“I always wanted to be on the cutting edge of treatments to try and haveas normal a life as possible.” – David Novak.

Today on Beyond the Bleeds:

• Hemophilia A is a genetic disorder characterized by a deficiency in factor VIII, necessary for blood clotting.
• Gene therapy for hemophilia A involves using a modified virus to deliver a working copy of the factor VIII gene to liver cells.
• Traditional treatments require infusions multiple times a week, while gene therapy aims to reduce or eliminate this need.
• Candidates for gene therapy must be adult males with severe hemophilia A, no active infections, and no antibodies to the specific vector virus.
• Pre-treatment requirements include extensive lab tests and liver health checks.
• Post-treatment care includes regular blood draws, monitoring of factor VIII levels, and liver health.
• Patients undergoing gene therapy need to adopt healthier lifestyles, including no alcohol and smoking, for at least a year post-treatment.
• Gene therapy isn’t labeled as a cure but aims to significantly reduce the annual bleeding rate and improve patients’ quality of life.

Resources Mentioned:

BioMarin: https://www.biomarin.com/

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