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

Testing is difficult for drugs for rare diseases, and new rules may make it harder for sufferers to obtain life-saving drugsUS 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. Continue reading...