Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
Bioblast Pharma Ltd.
Age
18–80
Sex
ALL
Healthy volunteers
Not accepted
You may be eligible if
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
You may not be if
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
