A significant milestone for the gene therapy last this week.

The FDA has granted accelerated approval to the first-ever gene therapy targeting the underlying cause of Glycogen Storage Disease Type Ia (GSDIa) — an ultra-rare metabolic disorder that currently requires patients to follow a relentless, round-the-clock cornstarch regimen just to avoid dangerous drops in blood glucose.

In this pivotal Phase 3 study, treated patients saw a significant reduction in their cornstarch requirements compared to placebo, offering real hope of easing a burden that has shaped daily life for patients and families for decades.

Read the full announcement here.