Since two groundbreaking treatments were approved in late 2023, only a handful of patients have been able to access the multimillion dollar, medically-intensive therapies.

Summary

Wedam Minyila, 19, is among the first to undergo a newly approved gene therapy for sickle cell disease, a condition affecting over 100,000 people in the U.S.

The $2.3M-$3.1M treatment, using CRISPR technology, offers a potential cure but involves intensive chemotherapy and significant risks.

Few patients have accessed it due to high costs, limited hospital capacity, and insurance hurdles.

The therapy represents a groundbreaking shift, promising relief from severe pain and improved quality of life for patients.