The legal pathway for experimental, ultra-rare disease treatments is rocky and expensive

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Dec 19, 2023 - 18:00
The legal pathway for experimental, ultra-rare disease treatments is rocky and expensive

Want to stay on top of the science and politics driving biotech today? Sign up to get our biotech newsletter in your inbox.

Today, we see a small biotech on the verge of ending a rare-disease program thanks to FDA difficulties  — and how that impacts patients. We fete the best of this year’s biopharma CEOs (hint: GLP-1s), and talk about post-doc and patient advocacy reform.

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