You are not alone.

At UK HealthCare, we have made the care and treatment of patients with complex diseases part of our mission.

If you or your child has been diagnosed with a rare or complex disease, we understand that this is not the beginning of your journey. You have likely been seeking answers for some time, and a diagnosis of a disease with few if any available treatments is daunting at best. It is our goal to offer you a medical home, where you can get the personalized treatment and answers you need, and where you may even help find answers for others like you.

For some of these conditions, such as those listed below, there exist promising new treatments, like gene therapy, to dramatically improve or cure the disease.

Our multidisciplinary approach to care means you'll have a team of experts on your side.

If this sounds right for you or your loved one, or you would just like to speak to someone to get more information, fill out this simple form. Someone will get back to you in no more than three business days.

What are novel, rare, orphan or gene therapies?

• Novel therapies are new and innovative treatments that are a significant improvement over existing options. These therapies often target diseases or conditions that have limited or no effective treatments, offering new hope for patients. 

• A rare disease is a medical condition that affects a small portion of the populations. A rare disease affects fewer than 200,000 people in the United States at a given time. 
• An orphan disease is a type of rare disease with a special status from the government to encourage the discovery of new therapies that otherwise may be neglected due to limited potential for profit. 
• Gene therapies are treatments that modify a patient's genetic information to treat or prevent a disease. These therapies can add, change or silence genes within cells to fix genetic defects, fight diseases or restore normal function.

Conditions We Treat and Potential Novel Medication Therapies

*List not inclusive. If you do not see a therapy here that you are looking for, please fill out the contact form above and someone will get back with you.

• Amyotrophic Lateral Sclerosis
  • Qalsody®
• Duchenne Muscular Dystrophy (DMD)
  • Elevidys
• Hemophilia
  • Hemgenix®
  • Roctavian^TM
  • Beqvez^TM
• Spinal Muscular Atrophy
  • Zolgensma®
  • Spinraza®
• Cancer
  • Bladder Cancer
    • Adstiladrin®
    • Anktiva®
  • CAR-T Cell therapy:
    • Abecma®
    • Breyanzi®
    • Yescarta®
    • Kymriah®
    • Tecartus®
    • Carvykti®
  • BiTE and other specialized infusion therapies
    • Epkinly®
    • Blincyto®
    • Kimmtrak®
    • Tecvayli®
    • Talvey®
    • Elrexfio®

Our Services

This is a challenging journey, but at UK HealthCare, you do not have to make that journey alone.

We know that in the management of complex diseases, an expert and experienced provider is just the beginning. It requires a multidisciplinary team of qualified professionals and services to help meet your specific needs. It also requires access to the targeted and/or novel therapies that may hold the answers you seek. 

UK Specialty Pharmacy & Infusion Services 

Not only is UK HealthCare proud to offer experts in a growing number of rare and/or complex diseases, we are home to a Specialty Pharmacy that was the FIRST in the nation to earn certification in the care and treatment of individuals with rare diseases from URAC, a prestigious accrediting organization.

Learn more about UK Specialty Pharmacy & Infusion Services. 

Learn more about Kentucky Children's Hospital. 

Learn more about Kentucky Neuroscience Institute.

Learn more about the UK Hemophilia Treatment Center.

Learn more about UK Markey Cancer Center.