Hemophilia Patient Receives FDA Approved Gene Therapy Treatment Likely To Eliminate The Need For Preventative Factor Replacement
July 31 2024 - 4:00AM
Cure 4 The Kids Foundation (C4K) announced it has completed
Nevada’s first successful infusion of Hemgenix®, a gene therapy
option for adult patients with hemophilia B — a genetic bleeding
disorder in which people do not produce a protein needed to create
blood clots.
Depending on severity, a hemophilia B patient can require
multiple infusions of factor IX on a weekly basis to prevent a
bleed. However, after the one-time Hemgenix® infusion, the gene
therapy is expected to enable the individual’s body to produce
their own Factor IX, eliminating weekly infusions.
Under the medical supervision of Dr. Aimee
Foord, director of C4K’s bleeding and clotting clinic
along with Dr. Joseph Lasky, medical director of
C4K and the entire C4K medical team, the infusion was completed in
C4K’s infusion suite.
The patient, a 39-year-old male from Arizona, will be monitored
on a regular basis to ensure the treatment’s success.
“This treatment is ground-breaking for a number of reasons,”
said Annette Logan-Parker, chief innovation and
advocacy officer at C4K. “Most importantly, Hemgenix® gives the
patient a freedom from daily or weekly factor infusions they have
not had since their original hemophilia diagnosis. From a larger
perspective, being approved to provide this life-changing treatment
is another example of how Cure 4 The Kids Foundation is bringing
innovation and cutting-edge therapies to the patients of Nevada and
the western United States.”
Hemgenix® (etranacogene dezaparvovec-drlb) is the first gene
therapy for the treatment of adults with hemophilia B who currently
use factor IX prophylaxis therapy, or have current or historical
life-threatening bleeding, or have repeated, serious spontaneous
bleeding episodes. Although Hemgenix® is not a cure for
hemophilia B patients, the medical advancement may reduce, if not,
eliminate weekly preventative factor replacement and bleeds.
Hemgenix® relies upon a gene transfer that is administered
through intravenous (IV) infusion. The approach introduces a
“working” or functional gene into liver cells to “instruct them” to
produce factor IX protein, which is deficient in patients with
hemophilia B, and which helps form blood clots to prevent
prolonged, excessive or life-threatening bleeding.
Hemgenix® has the potential to fundamentally transform the
treatment paradigm through a single, one-time infusion in people
living with hemophilia B by addressing the genetic cause of the
condition.
Because the genes that cause hemophilia A and B are located on
the sex-determining X chromosome, the disorder disproportionately
affects males. According to the U.S. Centers for Disease Control
and Prevention (CDC), as many as 33,000 males in the United States
are living with hemophilia. Hemophilia B is a rarer form of the
disorder, found in just 3.7 cases per 100,000 males.
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About Cure 4 The Kids FoundationFounded in Las
Vegas in 2007, Cure 4 The Kids Foundation provides high-quality,
research-focused medical treatment to children battling cancer and
other life-threatening conditions. Cure 4 The Kids Foundation
operates the only outpatient childhood cancer treatment center in
Nevada and is proudly accredited by The Joint Commission. This
stringent medical accreditation and the required unannounced
inspections ensure patients are getting the safest care possible. A
501(c)(3) nonprofit organization, Cure 4 The Kids Foundation
increases access to specialized treatments that improve patient
outcomes. Its Charity Care Program provides high-quality treatment
on a sliding-scale basis. No patient is ever turned away for
financial reasons. For more information, visit
www.cure4thekids.org.
- Cure 4 The Kids Foundation
- Cure 4 The Kids Foundation
Mitch Truswell
Cure 4 The Kids Foundation
7025314461
mitch@red7communications.com