Fanconi renotubular syndrome
MONDO:0001083A genetic or acquired disorder characterized by impairment of the function of the proximal tubules of the kidney. It results in decreased reabsorption of electrolytes, glucose, amino acids, and other nutrients.
Also known as: De toni-debre-Fanconi syndrome, Fanconi syndrome, Fanconi's syndrome, Fanconi-de toni syndrome, Lignac-Fanconi syndrome, adult Fanconi syndrome, congenital Fanconi syndrome, infantile nephropathic cystinosis
4 clinical trials for this condition and its sub-types, 4 tagged with Fanconi renotubular syndrome itself.
Follow this condition to get notified about new trialsWhere it sits in the disease tree
Browse by category →Part of
Sub-types of Fanconi renotubular syndrome
-
Deal Barratt Dillon syndrome 0 trials
-
Acquired Fanconi syndrome 0 trials
1 sub-type
- Preeyasombat-Varavithya syndrome 0 trials
-
Adult Fanconi syndrome 0 trials
-
3 sub-types
Most studied deeper sub-types
-
Gene therapy for rare blood disease passes 15-Year safety watch
Disease control OngoingThis study follows 9 people with Fanconi Anemia who already received a gene therapy that adds a working FANCA gene to their blood stem cells. Researchers will check their health and blood counts for 15 years to see if the treatment remains safe and keeps working. No new treatment…
Sponsor: Rocket Pharmaceuticals Inc. • Aim: Disease control
Last updated Jun 27, 2026 12:07 UTC
-
Experimental gene therapy aims to stop bone marrow failure in kids with rare disease
Disease control OngoingThis phase 2 trial tests a gene therapy called RP-L102 for children with Fanconi anemia subtype A, a rare genetic disorder that leads to bone marrow failure. Doctors take the child's own blood stem cells, fix the faulty gene in a lab, and infuse the corrected cells back. The goal…
Phase 2 • Sponsor: Rocket Pharmaceuticals Inc. • Aim: Disease control
Last updated Jun 27, 2026 08:06 UTC