Center for Rare Disease Therapy Providers and Staff

Meet our internationally-renowned experts who offer innovative therapies and specialized care for patients with rare diseases.

Gerard Vockley

Jerry Vockley, MD, PhD
Director, Center for Rare Disease Therapy
Chief, Division of Genetic and Genomic Medicine

Dr. Vockley is an international leader in treatment and research in genetic and genomic medicine and the field of inborn errors of metabolism. He is also a founder of the North American Metabolic Academy.

Steven Allen, MD

Steven Allen, MD
Pediatric Hematologist - Oncologist
Medical Director, Bone Marrow Failure Disorders

Dr. Allen quickly found his niche at UPMC Children's Hospital with the Center for Rare Disease Therapy, specializing in patients with bone marrow failure syndromes and histiocytic disorders.

Hey Jin Chong, MD, PhD

Hey Jin Chong, MD, PhD
Chief, Division of Pediatric Allergy and Immunology

Dr. Chong’s background in immunology enables her to draw on a network of researchers and specialized labs around the country, who assist her diagnostic work by performing highly sophisticated laboratory and genomic tests.

Kate Ellery, DO

Kate Ellery, DO
Pediatric Gastroenterologist
Medical Director, Pancreatic Disorders Center

Dr. Ellery is the medical director of the Pancreatic Disorders Center at UPMC Children’s, which is one of the few pediatric hospitals in the nation to offer patients total pancreatectomy with autologous islet-cell transplantation (TPAIT), a novel procedure designed to offer pain relief while greatly reducing a patient’s risk of developing diabetes.

Brian Feingold, MD

Brian Feingold, MD
Medical Director, Pediatric Heart Failure and Heart Transplant Programs

Dr. Feingold is an internationally recognized expert in the care of children with Barth syndrome, an extremely rare condition in which a cardinal symptom is heart failure.

Ira Fox, MD

Ira Fox, MD
Director, Center for Innovative Regenerative Therapies
UPMC Children's Hospital of Pittsburgh and McGowan Institute 
for Regenerative Medicine

A world authority on liver stem cells with over 30 years of experience in performing liver transplants, Dr. Fox is the first person to successfully transplant hepatocytes into a patient with a metabolic disorder.

George V. Mazariegos, MD, FACS Chief, Pediatric Transplantation

George V. Mazariegos, MD, FACS

Dr. Mazariegos and his team developed the protocol for liver transplantation for maple syrup urine disease and pioneered the use of liver transplantation as a therapeutic modality for metabolic disease. His research interests include transplant tolerance, immunosuppression withdrawal and optimizing long term transplant outcomes.

Patrick McKiernan, MD, Director, Pediatric Hepatology Program

Patrick McKiernan, MD
Director, Pediatric Hepatology Program

Dr. McKiernan is an internationally-renowned pediatric liver expert and specializes in the treatment of children with inherited metabolic disease.  He is actively involved in research on stem cell therapy for metabolic liver disease.

Julia Meade, MD

Julia Meade, MD
Pediatric Hematologist-Oncologist

Dr. Meade directs the Pediatric Cancer Predisposition Program, which is the only one of its kind in western Pennsylvania. She is responsible for the assessment, management and diagnosis of cancer predisposition syndromes in children. Her research interests include the patient and provider experience with family cancer syndromes.

Mousumi Moulik, MBBS

Mousumi Moulik, MBBS
Director, Pediatric Cardiovasular Genetics Clinic

An international expert in cardiovascular genetics, Dr. Moulik also directs a research lab where she and colleagues are working to unravel the genetics of cardiomyopathy and understand how the disease originates and develops.

Ken K. Nischal, MD, FRCOphth Chief, Division of Pediatric Ophthalmology, Strabismus, and Adult Motility

Ken K. Nischal, MD, FRCOphth
Chief, Division of Pediatric Ophthalmology, Strabismus, and Adult Motility

A pioneer in pediatric ophthalmic surgery, Dr. Nischal has developed surgical techniques for children with rare diseases to help cure the blindness sometimes seen in these conditions. Dr. Nischal has authored more than 100 published research articles and is one of the world’s foremost pediatric eye specialists, treating patients from all over the world.

Deepa Rajan, MD

Deepa Rajan, MD
Co-Director, Neuro-genetics Clinic

Dr. Rajan sees patients who have often severe neurologic and other symptoms that have so far eluded diagnosis, as well as patients who have been diagnosed with rare neurologic conditions caused by genetic abnormalities.

Kyle Soltys, MD

Kyle Soltys, MD
Pediatric Transplant Surgery

Dr. Soltys is an expert in complex liver transplant surgery, especially for patients who are diagnosed with rare metabolic diseases. He has been instrumental in developing partnerships with the Clinical for Special Children in Strasburg, Pa., and the University of Virginia Children’s Hospital in Charlottesville.

James Squires, MD, MS

Jim Squires, MD, MS
Pediatric Hepatology Program

Liver transplantation can be a pathway to a cure for several pediatric metabolic disorders. Dr. Squires is an expert in helping patients, parents, and caregivers to determine their best treatment option.

Kevin A. Strauss, MD
Medical Director, Clinic for Special Children

Dr. Strauss's expertise is in the application of knowledge from the rapidly evolving fields of genetic and genomic research to prevent disabilities in children caused by genetic diseases.

Paul Szabolcs, MD

Paul Szabolcs, MD
Chief, Bone Marrow Transplantation and Cellular Therapies

A pioneer in reduced-toxicity/intensity unrelated donor cord blood transplantation for inborn errors, Dr. Szabolcs is the first to demonstrate feasibility of sequential lung and bone marrow transplantation from the same unrelated cadaveric donor.