COVID-19 Vaccine Information and Updates Read the Latest

Affiliated Sites

Children's Hospital is part of the UPMC family.

  • UPMC Website
  • UPMC's Story

Our Sites

Foundation Logo
Give to Children's
CCP Logo
UPMC Children's Community Pediatrics
UPMC Logo
UPMC
UPMC Magee-Womens Hospital
UPMC Magee-Womens Hospital
  • International Patients
  • Careers
  • News
UPMC Children's Hospital of Pittsburgh
  • For Parents
  • Our Services
  • For Health Care Professionals
  • Research
  • About Us
Before Your Visit
  • Welcome Center
  • Accommodations
  • Visitor Information
  • Directions & Parking
  • Find more information about planning a visit »
During Your Visit
  • Contact a Patient
  • Hospital Amenities
  • Family Support Services
  • Getting Around Children's
  • Learn more about support and resources »
After Your Visit
  • Praise a Staff Member
  • Share Your Story
  • Medical Records
  • Billing
Injury Prevention
Health Library Image

Be safe anytime, anywhere.

Learn about safety »
Clinical Services
  • Asthma Center
  • Brain Care Institute (Neurology & Neurosurgery)
  • Cancer
  • UPMC Children's Express Care
  • Ear, Nose & Throat (ENT)
  • Emergency Medicine
  • Endocrinology
  • Gastroenterology
  • Heart Institute (Cardiology & Cardiac Surgery)
  • Infectious Diseases
  • Medical Genetics
  • Nephrology
  • Newborn Medicine
  • Primary Care
  • Pulmonary Medicine
  • Rheumatology
  • Surgery
  • Transplant Programs
  • Browse or search our full list of services »
Find a Doctor

To find a pediatrician or pediatric specialist, please call 412-692-7337 or search our directory.

Search Find a Doctor Directory »
For Physicians
  • Alumni Association
  • Make A Referral
  • Phone Directory of Services
  • Physician Liaisons
  • Webinar Series
  • View all »
Continuing Medical Education
  • Pediatric Grand Rounds
  • Rangos Research Seminars
  • Other CME Opportunities
  • View more »
Education & Training
  • Residencies
  • Fellowships
  • Medical Student Education Program
  • See more »
Pediatric INSIGHTS
Pediatric INSIGHTS Magazine

A resource for our network of referring physicians.

Read more »
Clinical Studies
  • Adolescent Medicine Studies
  • Cardiology Studies
  • Intestine Transplant Studies
  • Type 1 Diabetes Studies
  • Find more clinical studies »
Our Facilities
  • The Pediatric Clinical and Translational Research Center (PCTRC)
  • John G. Rangos Research Center
  • Learn more about our facilities »
Research Areas
  • Anesthesiology
  • Cardiology Research
  • Endocrinology Research
  • Genetics Research
  • View all research areas »
Contact the Research Department

For more information about research, please call our main office at 412-692-6438. 

Find additional contact information »
About Children's
  • Vision, Mission, & Values
  • Our History
  • Our Campus
  • Executive Leadership
  • Awards & Recognition
Find it Fast
  • Careers
  • Locations
  • Newsroom
  • Connect with Us
Additional Resources
  • Volunteering at Children's
  • Advocacy
  • Injury Prevention
  • myCHP
U.S. News & World Report | Best Children’s Hospitals

Ranked #9 Nationally by U.S. News & World Report.

Learn more »
  • Home
  • Our Services
  • Center for Rare Disease Therapy
  • Conditions We Treat
Center for Rare Disease Therapy
  • About Us
  • IDDAT Program
  • Conditions We Treat
    • Adrenoleukodystrophy
    • Alpha-1 Antitrypsin Deficiency
    • Bare Lymphocyte Syndrome (BLS)
    • Bone Marrow Failure Disorders
    • Byler Disease
    • CANDLE Syndrome
    • Chronic Granulomatous Disease (CGD)
    • Chronic Pancreatitis & Acute Recurrent Pancreatitis
    • Combined Immunodeficiency (CID)
    • Common Variable Immune Deficiency (CVID) Syndrome
    • Fabry Disease
    • Fatty Acid Oxidation Deficiencies
    • Gaucher Disease
    • Glutaric Acidemia Type 1
    • Glycogen Storage Disorders
    • GM3 Synthase Deficiency
    • Hemophagocytic Lymphohistiocytosis (HLH)
    • Hunter Syndrome (MPS II)
    • Hurler Syndrome (MPS I)
    • Krabbe Disease
    • Maple Syrup Urine Disease (MSUD)
    • Metachromatic Leukodystrophy (MLD)
    • Methylmalonic Acidemia
    • Mitochondrial Disorder
    • Niemann-Pick Disease Type C
    • Phenylketonuria (PKU)
    • Pompe Disease
    • Progressive Familial Intrahepatic Cholestasis
    • Propionic Acidemia
    • Severe Combined Immune Deficiency (SCID)
    • Urea Cycle Disorders
    • Vanishing White Matter Disease
  • Doctors & Staff
  • Patient Stories
  • Webinar Series
  • News & Events
  • Contact Us

Rare Diseases and Disorders We Treat

The Center for Rare Disease Therapy at UPMC Children's Hospital of Pittsburgh offers novel therapeutic modalities and personalized services to treat infants, children, adolescents, and young adults who are diagnosed with a variety of rare disorders, including:

  • Adrenoleukodystrophy
  • Alpha-1-Antitrypsin (AT) Deficiency
  • Bare Lymphocyte Syndrome (BLS)
  • Byler Disease
  • CANDLE Syndrome
  • Chronic Granulomatous Disease (CGD)
  • Chronic Pancreatitis and Acute Recurrent Pancreatitis
  • Combined Immune Deficiency (CID) Syndromes
  • Combined Variable Immune Deficiency (CVID) Syndrome
  • Congenital Corneal Opacity (Peters Anomaly and Sclerocornea)
  • Crigler-Najjar syndrome 
  • Fabry Disease
  • Fatty Acid Oxidation Deficiencies
  • Gaucher Disease
  • Glutaric Acidemia Type 1
  • Glycogen Storage Disorders
  • GM3 Synthase Deficiency
  • Hemophagocytic Lymphohistiocytosis (HLH) Syndromes
  • Hereditary Telangiectasia (HHT)
  • Hunter Syndrome (MPS-II)
  • Hurler Syndrome (MPS-I)
  • Krabbe Disease
  • Maple Syrup Urine Disease (MSUD)
  • Metachromatic Leukodystrophy
  • Methylmalonic Acidemia
  • Mitochondrial Disorder
  • Niemann-Pick Disease Type C
  • Phenylketonuria (PKU)
  • Pompe Disease
  • Progressive Familial Intrahepatic Cholestasis
  • Propionic Acidemia
  • Severe Combined Immune Deficiency (SCID)
  • Urea Cycle Disorders
  • Vanishing White Matter Disease

Meet the international experts of the Center for Rare Disease Therapy.

Accolades
U.S. News & World Report | Best Children’s Hospitals Magnet Recognized Hospital by the ANCC Parents Most Innovative Children's Hospitals LeapFrog Group's Top Children's Hospital
  • Find a Doctor
  • Search Locations
  • Contact Us
  • Find a Job
  • Health Library
  • Get Directions
  • Contact a Patient
  • Pay My Bill
  • © 2021 UPMC
  • UPMC'S MEDICAL ADVICE DISCLAIMER
  • UPMC Privacy Information
  • UPMC's Website & Email Terms of Use Policy

Supplemental content provided by Healthwise, Incorporated. To learn more, visit Healthwise.org.

Find a Doctor
Locations & Directions
Contact Us
myCHP
Pay My Bill
Donate

Search by:

Last Name

Preferences

Gender
Language
Role
Locations & Directions

Children's Hospital's main campus is located in the Lawrenceville neighborhood. Our main hospital address is:

UPMC Children’s Hospital of Pittsburgh
One Children’s Hospital Way
4401 Penn Ave.
Pittsburgh, PA 15224

  • Get directions to our main campus.

In addition to the main hospital, Children's has many convenient locations in other neighborhoods throughout the greater Pittsburgh region. 

  • Search our locations. 

Contact Us

  • For general information and inquiries, please call 412-692-5325.
  • To make an appointment, please call 412-692-7337 from 7 a.m. to 7 p.m. Monday through Friday.
  • Share a comment, compliment or concern.
  • Tell us what you think about our website - send an email to feedback@chp.edu.

myCHP: Manage your child's health information online - on your time!

With myCHP, you can request appointments, review test results, and more.

  • Log-In to myCHP
  • Sign Up: Parents, legal guardians, and patients may sign-up online. Parents, legal guardians, and patients may also sign-up in person during a hospital stay, at a clinic appointment, or by visiting the UPMC Health Plan Connect Service and Sales Center at your local mall. Learn More.

Pay My Bill

Hospital Billing

For questions about a hospital bill call:

  • UPMC Patient Financial Services Center: 1-800-371-8359
  • UPMC Customer Service: 1-844-591-5949

Online Bill Payment

To pay your bill online, please visit UPMC's online bill payment system.

Donate

Children's Hospital of Pittsburgh Foundation

Interested in giving to Children's Hospital? Support the hospital by making a donation online, joining our Heroes in Healing monthly donor program, or visiting our site to learn about the other ways you can give back.