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Facio Scapulo Humeral Muscular Dystrophy (FSHD)

Apabetalone has been shown to inhibit the expression of DUX4, the protein identified as the primary cause of FSHD, in human skeletal muscle cells. Apabetalone is expected to soon begin testing in an FSHD rodent model of disease. At the same time, R&D efforts continue to optimize the muscle-specific delivery of the drug.

Primary Endpoint

Time from randomization to the first occurrence of adjudication-confirmed Major Adverse Cardiac Event (MACE) defined as a single composite endpoint of: 1) Cardiovascular death, or 2) Non-fatal myocardial infarction, or 3) Stroke.

Primary Objective

To evaluate if treatment with apabetalone as compared to placebo increases time to the first occurrence of a Major Adverse Cardiac Event (MACE). MACE is defined as a single composite endpoint of: 1) Cardiovascular death, or 2) Non-fatal myocardial infarction, or 3) Stroke.