Hartford Mother Fights for FDA Approval of Drug to Help Son with Rare Genetic Illness

Hartford mother fights for FDA approval of drug to help son with rare genetic illness

A Hartford mother is pushing for U.S. Food and Drug Administration approval of a drug that could help her 13-year-old son who lives with a rare genetic illness.Amy Wald emailed TMJ4 News asking to share her son Levi's story of survival. He is living with Barth Syndrome, a genetic illness caused by a random mutation that affects his breathing, heart and muscle development.According to Amy, only 200 people live with this illness across the world.W…

Rankin County teen living with rare disease awaits FDA approval for drug he says changed his life

Rankin County teen living with rare disease awaits FDA approval for drug he says changed his life RANKIN COUNTY, Miss. (WLBT) – The Food and Drug Administration is holding a drug in the balance that one family says improves the lives of those with rare diseases. One Rankin County family says the medication Elamipretide has given one teenager the strength to be a normal kid, and they don’t want that taken away. “It’s helped a lot. Before, I could…

11-year-old boy with rare genetic kidney disease looking for donor

Blake Shore was born with a rare genetic disease and also has polycystic kidney disease. Because of the disease, he now needs a new kidney.