Parent of Terminally Ill Child Invites FDA Commissioner to Witness Impact of Regulatory Delays
Updated
Updated · Fox News · Apr 29
Parent of Terminally Ill Child Invites FDA Commissioner to Witness Impact of Regulatory Delays
9 articles · Updated · Fox News · Apr 29
Ryu, a 14-year-old with Duchenne muscular dystrophy, faces $30,000 monthly steroid costs and relies on experimental drugs inaccessible due to FDA review delays.
The parent urges Commissioner Makary to visit their home, highlighting how regulatory caution and extended review cycles have dashed hopes for timely treatment and worsened quality of life.
With the upcoming departure of FDA’s biologics chief, families call for leadership that prioritizes patient involvement and aligns regulatory timelines with the urgent needs of rare disease patients.
Should a parent’s desperate plea override the FDA's scientific process for approving experimental drugs for their child?
If 'Expanded Access' to unapproved drugs exists, why is it not a realistic solution for most rare disease patients?
With a controversial FDA director leaving, will his successor finally fix the 'moving goalposts' for rare disease drugs?
An approved Duchenne therapy had safety issues. How will new experimental treatments be proven safer before approval?
A mother says doing nothing is deadly, but are we ignoring the risks of rushing unproven gene therapies?