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Sulfamethoxazole treatment in primary PREPL deficiency
2013-005475-41Phase 2Completed
- Sponsor
- UZ Brussel
- Start
- May 2014
- Modality
- Small Molecules
- Countries
- 1
01
Overview
Phase 2, single-blind, single-arm trial of sulfamethoxazole in PREPL deficiency and Hypotonia-Cystinuria Syndrome (HCS), sponsored by UZ Brussel. Primary endpoint: Ptosis evaluated with the MG composite. Started May 2014; status: completed.
02
Drugs
sulfamethoxazole
03
Conditions
PREPL deficiencyHypotonia-Cystinuria Syndrome (HCS)
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