The Calliope Joy Foundation raises funds to help children living with leukodystrophy through improved care, research, and family support. Join us for an evening with Ron Jaworski and friends to benefit the Leukodystrophy Center of Excellence at the Children’s Hospital of Philadelphia and The Calliope Joy Foundation.
The Leukodystrophy Center of Excellence at Children’s Hospital of Philadelphia (CHOP) provides comprehensive clinical care, diagnostic testing and the most advanced treatments available to infants, children and adolescents with inherited white matter diseases. The Center’s research leads to clinical trials, new treatments and potential cures. Research studies led by neurologists Adeline Vanderver, MD, Amy Waldman, MD, and Laura Adang, MD, PhD are pivotal in understanding disease mechanisms and the impact of the leukodystrophies on a child’s health and quality of life. Gene therapies, enzyme replacement, or other means to address these genetic disorders bring hope to the horizon.
Since 2015, the Calliope Joy Foundation has been instrumental in supporting early phase pre-clinical development of therapies for Aicardi-Goutieres syndrome (AGS), H-ABC and metachromatic leukodystrophy (MLD), as well as enabling the care of the children and adults with leukodystrophy via standards of care and support for CHOP’s Leukodystrophy Center. The amazing successes of the Center are evident in the increasing number of genetic causes discovered, which brings identity and ends the diagnostic mystery that so many families embark on for far too long.
Funds raised from this year’s Cupcake Gala will directly support our ongoing research in molecular and gene therapy for the leukodystrophies and patient care.