this post was submitted on 06 Jul 2025
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Patients with ultra-rare diseases worry new FDA rules will leave them without treatment (www.theguardian.com)
submitted 2 days ago by babysandpiper@sopuli.xyz to c/health@lemmy.world
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Testing is difficult for drugs for rare diseases, and new rules may make it harder for sufferers to obtain life-saving drugs

US drug regulators 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 clinical trials.

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.

Patients and advocates worry about new rules on who may receive the medication during this process, and whether the drug will reach approval before the pharmaceutical company runs out of funding for it.

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[–] imrighthere@lemmy.ca 7 points 2 days ago

Of course it will, that is the point. yarvin made it crystal clear that the idea was to kill off a ton of people. They told you this right to your faces. The good news is, you're going to wave signs at nazis twice a month. Problem solved.