Big Take23 Joulu 2022

Declining ALS Patients Are Waiting On The FDA's Next Move

For many years, pharmaceutical companies have tried and failed to find a treatment to slow symptoms of ALS–the debilitating, fatal illness also known as Lou Gehrig’s Disease. A potentially promising new drug from Biogen may offer some relief to those afflicted with an uncommon and especially insidious form of ALS that runs in families. Patients are urging the US Food and Drug Administration to speed access to the drug, which hasn’t yet been approved because clinical trials didn’t conclusively show it works.

As pharmaceutical companies use new technologies to seek cures of all kinds, this tension between making sure drugs are effective and safe, and offering not-quite-ready but possibly life-saving treatment to people for whom it’s now or never, is becoming more acute.

Bloomberg’s chief medical writer Robert Langreth joins this episode to talk about the race to find a treatment for ALS–and the back and forth between patients, companies and government regulators. We also hear the story of a Pennsylvania family that has passed on a gene for ALS from generation to generation–and their demands for access to the new treatment.

Learn more about the episode here: https://bloom.bg/3WIkvCm

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Have questions or comments for Wes and the team? Reach us at bigtake@bloomberg.net.

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