NEPHROPATHIC CYSTINOSIS

Clinical trials for NEPHROPATHIC CYSTINOSIS explained in plain language.

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Trials to join now! 1 Not yet recruiting 1 Not yet finished but already full! 1 Completed 1

Gene therapy may free kids from daily cystinosis meds

Disease control Recruiting now

This study tests a gene therapy called DFT383 in 30 children aged 2 to 5 with nephropathic cystinosis, a rare disease that damages kidneys and other organs. The treatment aims to fix the genetic problem so children may no longer need daily cysteamine medication. Researchers will …

Matched conditions: NEPHROPATHIC CYSTINOSIS

Phase: PHASE1, PHASE2 • Sponsor: Novartis Pharmaceuticals • Aim: Disease control

Last updated Jun 27, 2026 13:00 UTC

NEPHROPATHIC CYSTINOSIS

Get alerted when new NEPHROPATHIC CYSTINOSIS trials appear

Gene therapy may free kids from daily cystinosis meds