Patients with ultra-rare diseases worry new FDA rules will leave them without treatment

<p>Testing is difficult for drugs for rare diseases, and new rules may make it harder for sufferers to obtain life-saving drugs</p><p>US drug <a href="https://www.theguardian.com/business/regulators">regulators</a> have increasingly signaled a focus on faster approvals and rare diseases, but patients with ultra-rare ailments fear they are falling through the cracks, especially given challenges to conducting <a href="https://www.theguardian.com/science/medical-research">clinical trials</a>.</p><p>One drug, elamipretide, garnered a narrow recommendation from independent advisers for the US Food and Drug Administration (FDA), but the agency rejected the drug’s application in May and recommended another potential pathway for approval.</p> <a href="https://www.theguardian.com/science/2025/jul/06/fda-drugs-ultra-rare-diseases">Continue reading...</a>