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#genetic medicine

NIH Study Finds New Potential Approach for Cystic Fibrosis Treatment Health Care

NIH Study Finds New Potential Approach for Cystic Fibrosis Treatment

NIH researchers report a potential cystic fibrosis treatment using engineered tRNA and lip…

FDA Approves First Treatment for Rare MCT8 Deficiency Health Care

FDA Approves First Treatment for Rare MCT8 Deficiency

FDA approves the first treatment for MCT8 deficiency, a rare genetic disorder affecting th…

FDA Approves First Gene Therapy for Children With Sanfilippo Syndrome Health Care

FDA Approves First Gene Therapy for Children With Sanfilippo Syndrome

The FDA has approved Fayuvi, the first gene therapy for pediatric patients with Sanfilippo…


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