Posts by superadmin

Four years ago, my daughter Olivia was diagnosed with a rare and aggressive genetic disorder that attacks the brain and nervous system. She was rapidly losing the ability to walk and talk, and doctors told us that the disease was progressive, meaning Olivia’s condition would continue to worsen and she would eventually need hospice care. She was not yet 2 years old.

While reading Judy Stecker’s “The FDA Could Help Save My Son From a Rare Disease” (op-ed, Feb. 28), I couldn’t stop thinking about my own children’s battle against a rare disease—and the Food and Drug Administration red tape that kept effective treatment an ocean away.

A new gene therapy for the fatal genetic disorder metachromatic leukodystrophy, or MLD, will carry a wholesale price of $4.25 million, its manufacturer announced Wednesday, making it the world’s most expensive medicine.