Clinical trials: Isolated PREPL Deficiency
Hypotonia Cystinuria Syndrome, Isolated PREPL Deficiency
Sulfamethoxazole for the Treatment of Primary PREPL Deficiency
The investigators will evaluate whether sulfamethoxazole, a sulfamide antibiotic, improves the symptoms of primary PREPL deficiency (hypotonia-cystinuria syndrome and isolated PREPL deficiency).
- Trial name
- SPPD (NCT02640443)
- Testing
- Sulfamethoxazole
- Volunteers
- 15 people
- Sponsor
- Universitair Ziekenhuis Brussel