Gene therapies have the potential to cure some diseases, but they are extraordinarily expensive. Location can also be a big ...

A pioneering gene therapy could help treat a rare seizure disorder called Dravet syndrome, according to new clinical trial ...

While the FDA appears to be adamant that uniQure conduct a sham surgery–controlled Phase 3 trial before AMT-130 can be ...

A new clinical trial led in part by a Lurie Children’s Hospital doctor is showing promise for children with Dravet syndrome, ...

Lowering cholesterol is one of the most effective ways to reduce your risk of heart disease, and it may soon be possible to get a one-and-done gene therapy to keep cholesterol and triglyceride levels ...

Your heart will not just give you fear and uncertainty after a heart attack; it can leave scar tissue that stiffens your heart and limits your strength. Many people learn to live with that loss, but ...

A new class of drugs is saving the lives of children who once had no hope. These high-tech medicines can replace defective genes. But there's a catch. Many cost millions of dollars for a single dose.

CRISPR gene therapy Casgevy can nearly eliminate sickle cell crises, but difficult stem‑cell collection is delaying access ...

Successful program buildout prioritizes defined patient flow, infusion access, cold-chain capabilities, and accountability ...

When Harith Rajagopalan considers the millions of patients who have taken a GLP-1 drug to treat diabetes or obesity, he sees a revolution that is failing to realize its promise. “We are literally ...

Health system leaders face difficult choices when deciding how to build cell and gene therapy programs. These treatments can ...