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.
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.
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.
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.
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.
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.
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.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07778823 |
Augmented Reality-Cardiopulmonary Resuscitation Support for Pediatric Resuscitation |
RECRUITING | NA |
| NCT07743554 |
Combination of Avutometinib and Defactinib for Treatment of Relapsed, Refractory, Metastatic, or Unresectable Malignant Peripheral Nerve Sheath Tumor |
NOT_YET_RECRUITING | PHASE2 |
| NCT07718802 |
Observational Approach Versus Surgical Intervention for Stones |
NOT_YET_RECRUITING | NA |
| NCT07707037 |
Removing Surrogates Uncertainty to Reduce Fear and Anxiety After Cardiac Events - Kids |
RECRUITING | NA |
| NCT07687238 |
The Social Needs Assistance for Hospitalized Kids Trial |
NOT_YET_RECRUITING | NA |
| NCT07635030 |
GABA and GSH in FRDA |
ENROLLING_BY_INVITATION | |
| NCT07582211 |
Bilevel Positive Airway Pressure (BPAP) for Severe Asthma |
RECRUITING | NA |
| NCT07564583 |
Pilot Study of a Modified Safety Planning Intervention for Elementary School-Aged Children - Main Study |
ENROLLING_BY_INVITATION | NA |
| NCT07554560 |
Nutrition Intervention for Pancreatic Cancer |
RECRUITING | NA |
| NCT07549646 |
24VA021; VATCH Trametinib for Ras/MAPK Pathway VAs |
ACTIVE_NOT_RECRUITING | PHASE2 |
