Overview

Pharmacological Treatment of a Rare Genetic Disease: N-acetylcysteine in Myopathy Associated Selenoprotein N-related Myopathy (SEPN1-RM)

Status:
Terminated

Trial end date:
2020-08-31

Target enrollment:

Participant gender:

Summary

The objective of this study is to determine whether the administration of N-acetylcysteine (NAC) improves oxidative stress. To determine this, the study will assess the impact of oral treatment on the balance between reduced and oxidized form of glutathione in erythrocytes of peripheral blood.

Phase:

Phase 2/Phase 3

Details

Lead Sponsor:

Assistance Publique - Hôpitaux de Paris

Treatments:

Acetylcysteine
N-monoacetylcystine