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Aug 24, 2026
New noninvasive biomarkers are advancing early detection of fibrosis. A FAP-Index blood test reduces uncertain liver fibrosis results by up to 70%, a urine-based molecular imaging test detects kidney fibrosis with 84% sensitivity and 94% specificity, and liver MRE can track Gaucher disease severity over time.
May 08, 2026
Recent studies highlight progress in monoclonal antibodies against measles, Lyme disease, and Epstein-Barr virus. A Phase I Lyme antibody trial showed lasting protection, while preclinical antibodies blocked EBV and measles infection.
Jun 02, 2026
Gene editing repaired a Dravet syndrome mutation in mice, a prenatal hemophilia A therapy showed maternal safety in sheep, and researchers advanced toward a genetic test for valproic acid use in pregnancy.
May 05, 2026
The United Arab Emirates has unveiled two gene therapy projects targeting autism, epilepsy, and rare brain disorders in children. The initiatives focus on underlying genetic causes and earlier diagnosis.
Mar 11, 2026
The FDA has established a "plausible mechanism pathway" to approve personalized genome editing and RNA-based therapies for rare and ultra-rare diseases without requiring large randomized controlled trials, streamlining access to individualized treatments.
Feb 26, 2026
The FDA has proposed a new "plausible mechanism" pathway to approve customized treatments for rare diseases after testing in only a handful of patients, aiming to remove barriers for gene editing and other bespoke therapies that don't fit traditional approval systems.
Feb 24, 2026
The FDA has proposed new guidelines to create a pathway for approving bespoke therapies tested in small patient groups, specifically targeting rare genetic conditions and gene editing treatments that don't fit traditional approval systems.
