8-year-old walks again after experimental drug for rare condition
An 8-year-old boy, wheelchair-bound due to a rare genetic disease, HPDL deficiency, regained his mobility through an experimental drug, 4-HB. This groundbreaking treatment, bypassing the CoQ10 deficiency, restored his ability to walk and engage in activities like hiking and go-karting.
An 8-year-old boy, wheelchair-bound due to a rare genetic disease, HPDL deficiency, regained his mobility through an experimental drug, 4-HB. This groundbreaking treatment, bypassing the CoQ10 deficiency, restored his ability to walk and engage in activities like hiking and go-karting.
