PETOSKEY RESIDENT RECEIVES GENE THERAPY, HEMGENIX AT HEMOPHILIA OUTREACH CENTER IN GREEN BAY AND BECOMES THE FIRST MICHIGAN RESIDENT TO BE COMMERCIALLY DOSED.
PETOSKEY, MI- Petoskey resident with Hemophilia B is the first patient in the state to receive gene therapy, HEMGENIX, since FDA approval. “With this treatment and the promise of actually feeling even just a little better my hopes and dreams are already starting to become more attainable and desirable as my optimism returns to me. Better days lie ahead and I can only hope that everyone else in the community feels this same energy soon as we all deserve to live out these good days we might have told ourselves probably wasn’t in the cards.” – Andrew Bartkowiak, Patient of the Munson Health in Traverse City with Hemophilia B
“HEMGENIX is an important addition to the current treatment paradigm and a long-awaited advancement for the hemophilia B community,” said Matthew Ryan, MD, Adult Hematologist and Medical Director at Hemophilia Outreach Center, Green Bay, WI. “This exciting treatment offers patients a chance to live a life without frequent bleeds or the burden of regular infusions. The availability of this new one-time treatment also sparks additional conversations in the community when it comes to treatment options and goals, which is extremely important when managing a life-long condition, as an individual’s needs can change for a variety of reasons.”
“I’m excited that patients have this new opportunity that will lead to such a better quality of life versus their current prophylaxis treatment! To live a life free from recurrent prophylaxis, not having that anxiety over if they are going to have a bleed or if they have factor with them, not worrying about damage to their joints from breakthrough bleeds….it will be very lifechanging for them.”- Andrea Miller, APNP, Hemophilia Outreach Center.”- Andrea Buxton, APNP, Hemophilia Outreach Center.
The patient’s gene therapy infusion is scheduled for Wednesday, July 31st at the Hemophilia Outreach Center (HOC) in Green Bay. HOC is a family-oriented organization committed to providing the highest quality comprehensive medical care, and advocacy for individuals with a bleeding disorder. HOC serves approximately 400 patients in Northeastern and Central Wisconsin. Learn more at: https://hemophiliaoutreach.org/
HEMGENIX (etranacogene dezaparvovec), manufactured by uniQure Inc. and distributed by CSL Behring LLC, was FDA approved November of 2022 and is for the treatment of adults with Hemophilia B (Factor IX Deficiency) who currently use Factor IX preventative therapy, or have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes. Hemophilia B is a genetic bleeding disorder resulting from missing or insufficient levels of blood clotting Factor IX, a protein needed to produce blood clots to stop bleeding. Symptoms can include prolonged or heavy bleeding after an injury, surgery, or dental procedure; in severe cases, bleeding episodes can occur spontaneously without a clear cause. Prolonged bleeding episodes can lead to serious complications, such as bleeding into joints, muscles or internal organs, including the brain.
Although HEMGENIX is not a cure for Hemophilia B patients, the medical advancement may reduce, if not, eliminate weekly preventative factor replacement and bleeds.
