The primary cause of facioscapulohumeral muscular dystrophy (FSHD), a common adult-onset
dystrophy, was recently discovered identifying targets for therapy. As multiple drug
companies pursue treatments for FSHD, there is an urgent need to define the clinical trial
strategies which will hasten drug development, including creating disease-relevant outcome
measures and optimizing inclusion criteria. This proposal will develop two new outcome
measures and optimize eligibility criteria by testing 160 patients in 7 sites over a period
of 18 months.

The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics (PK)
of 5 escalating doses of SRP-5051 administered as a single dose to patients with DMD amenable
to exon 51 skipping treatment.

This is a multi-center, randomized, double-blind, placebo-controlled study to assess the
efficacy, safety and tolerability of two different weekly doses of BMS-986089 in ambulatory
boys with Duchenne Muscular Dystrophy (DMD).

Study A083-02 is a multicenter, Phase 2 study to evaluate the safety, tolerability,
pharmacodynamics (PD), efficacy, and pharmacokinetics (PK) of ACE 083 in patients with FSHD
to be conducted in two parts. Part 1 is open-label, dose-escalation and Part 2 is
randomized, double-blind, and placebo-controlled.

The main objective of this study is to evaluate the efficacy of SRP-4045 and SRP-4053
compared to placebo in Duchenne muscular dystrophy (DMD) patients with out-of-frame deletion
mutations amenable to skipping exon 45 and exon 53 respectively. Additional objectives
include evaluation of safety, pharmacokinetics and biomarkers.