NEPHROPATHIC CYSTINOSIS

Clinical trials for NEPHROPATHIC CYSTINOSIS explained in plain language.

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Trials to join now! 1

One-Time gene therapy aims to cure devastating childhood kidney disease

⭐️ CURE ⭐️ Recruiting now

This study is testing a one-time gene therapy called DFT383 in young children (ages 2-5) with nephropathic cystinosis, a rare and serious genetic disease that damages the kidneys and other organs. The main goals are to see if the treatment is safe and if it can reverse kidney dam…

Matched conditions: NEPHROPATHIC CYSTINOSIS

Phase: PHASE1, PHASE2 • Sponsor: Novartis Pharmaceuticals • Aim: ⭐️ CURE ⭐️

Last updated Mar 27, 2026 12:38 UTC

NEPHROPATHIC CYSTINOSIS

Get alerted when new NEPHROPATHIC CYSTINOSIS trials appear

One-Time gene therapy aims to cure devastating childhood kidney disease