Gene therapy aims to address the root cause of hemophilia B and help people produce stable and protective levels of...

What exactly is a gene? What happens when genes mutate? And can gene therapy research help solve problems related to...

Over 10,000 individuals worldwide have been treated with various gene therapy products. Gene therapy is a medical treatment that uses...

When Joseph Burke wakes up in the morning, his first question isn’t how he is going to feel today, it’s...

Randall Curtis, 66, survived life-threatening bleeds in the years before scientists discovered how to make clotting factor for home infusion.

Sponsored content by Sanofi Genzyme. Healthy joints are important for our health. They allow us to do the things in...

myWAPPS provides real-time factor level estimates, based on your pharmacokinetics (PK) profile

Pharmacokinetics (PK) can help you and your doctor determine the right dose and infusion frequency for you.

Explore pharmacokinetics (PK) terms and learn the role of PK in creating a treatment plan that matches your needs.

AscellaHealth’s groundbreaking hemophilia management program is designed with patient care at its core.

At uniQure, we know that people with genetic diseases like hemophilia B may have questions about immunity, antibodies, T cells...

You might have read our last few articles where we ask a lot of important questions about gene therapy, such...