Innovation in Gene Therapy Starts at UPMC Children’s
UPMC Children’s is on its way to becoming the lead site for development and human clinical trials of a new gene therapy treatment for VLCADD (Very Long Chain Acyl CoA Dehydrogenase Deficiency), a rare genetic disorder that is identified through newborn screening.
UPMC Children’s Chief of Genetic and Genomic Medicine Dr. Jerry Vockley recently received a grant from Kriya Pharmaceuticals that will empower him to develop the ground-breaking treatment. “Kriya reached out to me because we at UPMC Children’s are renowned for being able to develop and move new therapies into clinical trials,” Dr. Vockley shares.
VLCADD can lead to extremely weak heart and skeletal muscle, cardiorespiratory failure, and repeated bouts of low blood sugar, and it can be life-threatening in children if not treated. Dr. Vockley has already developed the only FDA-approved medication for VLCADD, and this grant will enable him to advance care to the next level.
Dr. Vockley’s research around VLCADD is one of numerous gene therapy research studies happening at UPMC Children’s. Gene therapy is an experimental treatment that aims to modify the genetic information of the patient’s disease-causing cells, thereby curing or lessening the severity the disease. It is a field considered by many to be the future of medicine.
So, why exactly is UPMC Children’s excelling in gene therapy? “We are such a strong clinical institution, and we have that translational mindset,” Dr. Vockley says. “We can do the research and treat the patients simultaneously, which makes us a great partner for pharmaceutical companies who want to pursue new treatments and therapies.”
The Arnold & Winnie Palmer Foundation Awards Grant to Further Cancer Research
Named for legendary golfer and Latrobe native Arnold Palmer and his wife, Winnie, the Arnold & Winnie Palmer Foundation is committed to making a difference in the lives of children and families.
Sydney was diagnosed with a rare genetic disorder called Urea Cycle Disorder / Ornithine Transcarbamylase deficiency (UCD/OTC) at just one year old.
World-Class Palliative Care for Every Child and Family, at Every Stage of Life
The new Division of Palliative Medicine and Supportive Care aims to build a future where there are no barriers to this life-changing care.
Lorenzo’s Journey to the Heart Institute at UPMC Children’s
Within days of his birth, a team of skilled surgeons at UPMC Children’s performed open heart surgery on Lorenzo.
Children’s Trust Grants $100,000 for Startup Pediatric Research
Each spring, a group of hand-selected researchers pitch next generation, budding research concepts to members of the Children’s Trust for a chance at winning seed funding to help kickstart their research.
Welcoming New Leadership on the Board of Trustees
Learn more about Vanessa and what inspires her to serve UPMC Children’s Hospital of Pittsburgh so proudly, in her own words.
Philanthropy Supports Individualized Behavioral Health Care
Throughout our hospital, there are many licensed psychologists dedicated to supporting specific service lines.