Archive for lysosomal storage disorders
Against all odds: Mila’s unique mutation, and her own custom drug
Ed. note: Mila passed away in February 2021, at age 10. The Mila’s Miracle Foundation continues to work to pave a pathway for personalized treatments. The FDA recently released a draft guidance on testing custom drugs such as Mila’s in patients. As a baby and toddler, Mila was healthy, active, and — in some ways ... Read More