This is the first ever gene therapy at UTMB.
From Dr. Erin Cooney
"Prior to recent advancements, a diagnosis of SMA type 1 would mean death before 2 years of age. This gene therapy is giving Blaise a chance at life. Dr. Lupo and I want to thank the entire clinical, pharmacy, and finance teams for making this special day happen. We couldn’t have done it without everyone’s hard work and commitment to this child."
TMC News highlights this extraordinary treatment and the team that made it happen—
Making muscles with the most expensive drug in the world (20 min read).