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 |
|---|---|---|---|
| NCT07582211 |
Bilevel Positive Airway Pressure (BPAP) for Severe Asthma |
NOT_YET_RECRUITING | NA |
| NCT07564583 |
Pilot Study of a Modified Safety Planning Intervention for Elementary School-Aged Children - Main Study |
ENROLLING_BY_INVITATION | NA |
| NCT07554560 |
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RECRUITING | NA |
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24VA021; VATCH Trametinib for Ras/MAPK Pathway VAs |
ACTIVE_NOT_RECRUITING | PHASE2 |
| NCT07544979 |
Creation of a Decision Aid for Coronary Anomalies |
NOT_YET_RECRUITING | NA |
| NCT07543822 |
24VA022; VATCH Alpelisib for TIE2/PIK3CA Pathway VAs |
ACTIVE_NOT_RECRUITING | PHASE2 |
| NCT07540767 |
Development of a Provider-Focused Intervention to Improve Health Outcomes in Pediatric Sickle Cell Disease |
NOT_YET_RECRUITING | NA |
| NCT07521657 |
Efficacy of Mirdametinib Alone or Combination With Radiotherapy for Germline and Sporadic NF1-Altered High-Grade Glioma |
NOT_YET_RECRUITING | PHASE2 |
| NCT07508436 |
Crisis Interventions for Pediatric Providers - Autism Version |
NOT_YET_RECRUITING | NA |
| NCT07501247 |
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NOT_YET_RECRUITING | NA |
