Conditions: Oculopharyngeal Muscular Dystrophy
Keywords: Case control, safety, tolerability, efficacy.
Allocation: NA | Intervention model: SINGLE_GROUP
Enrollment: 25
Healthy volunteers: no

Brief summary:
The Purpose of this study is to assess the Safety, Tolerability and Efficacy of Intravenous Cabaletta® in Oculopharyngeal Muscular Dystrophy (OPMD) Patients.

Detailed description:

Arms:
arm 1: weekly IV of Cabaletta 30gr.

Interventions: intervention 1: Cabaletta
Intervention descriptions: intervention 1: None

Protocol document: