Topic
#rare disease 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…
Health Care
FDA Approves First Treatment for Rare MCT8 Deficiency
FDA approves the first treatment for MCT8 deficiency, a rare genetic disorder affecting th…
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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