Safety Tolerability and Efficacy Study of Cabaletta to Treat Oculopharyngeal Muscular Dystrophy (OPMD) Patients

Inclusion Criteria:

- Males and females

- 18 - 80 years (inclusive) of age

- Clinically and genetically diagnosed as OPMD

- Moderate dysphagia (abnormal drinking test at screening and on the first dosing day,
before drug administration)

- Patients who provide written informed consent to participate in the study

- Body Mass Index (BMI) <30 kg/m2

Exclusion Criteria:

- Diabetes mellitus type 1 or 2

- Other major diseases, e.g.: renal failure (creatinine clearance <60ml/min), liver
failure and chronic liver diseases (e.g. hepatitis B or C) , HIV carriers,
tuberculosis, SLE, rheumatoid polyarthritis, sarcoidosis, collagenosis

- Uncontrolled heart disease , CHF,

- Other neuromuscular diseases

- Other disorders associated with esophageal dysphagia: e.g. severe gastroesophageal
reflux (GERD), esophageal stricture due to mechanical or chemical trauma, infection
(e.g. esophageal moniliasis), drug-induced dysphagia (e.g. bisphosphonates),
esophageal rings and webs, spastic motility disorders of the esophagus.

- History of malignancy (except non-invasive skin malignancy)

- History of neck irradiation

- Pregnant or currently lactating women

- Obesity (BMI≥ 30) and associated morbidity

- Prior pharyngeal myotomy

- Weight loss of more than 10% in the last 12 months.

- Known hypersensitivity to any ingredients in the injection

- Patient receiving anticoagulant treatment (e.g. warfarin)