Four years ago, my daughter Olivia wasย diagnosed with a rare and aggressive genetic disorder that attacks the brain and nervous system… A few months later, our youngest daughter, Keira, was diagnosed with the same genetic disorder โ metachromatic leukodystrophyย (MLD).
But unlike her older sister, Keira had not yet begun to show symptoms. That bit of luck gave us the extraordinary opportunity to try to save her life, as a cutting-edge gene therapy treatment was showing promising results in nonsymptomatic MLD patients.
There was one caveat: The five-month long, personalized treatment, which would be tailored to Keira based on her DNA, was not available in the United States due to the outdated U.S. drug approval process.
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Patients like my daughter do not have time to waste. Luckily, there is a solution that policymakers can implement to give patients hopeย now: theย Right to Try for Individualized Treatments.
This lawย would allow patients suffering from rare and genetic diseases to try personalized treatments not yet approved by the FDA, as long as they have the support of their physician and have exhausted other treatment options.This policy would have an outsized impact on patients with rare diseases. Although rare diseases have small patient pools by definition, collectively, aboutย 30 million Americansย are estimated to have a rare disease.
