Multi-Center Study of ManNAc for GNE Myopathy

Exclusion Criteria

• •1. Subject had an infection or medical illness requiring intravenous antibiotics or hospitalization within 30 days prior to the baseline/randomization visit.
• •2. Subject has another comorbid condition which may affect physical function.
• •3. Subject has a psychiatric illness or neurological disease that would interfere with the ability to comply with the requirements of this protocol.
• •4. Subject with hepatic laboratory parameters (AST, ALT, GGTP), equal to or greater than 3 times the upper limit of normal at screening.
• •5. Subject with existing renal dysfunction, as defined by glomerular filtration rate (GFR) less than 60 mL/min/1.73 m2 at screening.
• •6. Subject is anemic (defined as Hematocrit \<30%) or has platelets \<75 x 10\^3/µL or white blood cell count less than 3 x 10\^3/µL at screening.
• •7. Subject shows evidence of clinically significant cardiovascular, pulmonary, hepatic, renal, hematological, metabolic, or gastrointestinal disease, or has a condition that requires immediate surgical intervention.
• •8. Subject is pregnant or breastfeeding at any time during the study.
• •9. Subject has received treatment with another investigational drug, investigational device, or approved therapy for investigational use less than 90 days prior to screening.
• •10. Subject has received any dose of ManNAc, sialic acid, intravenous immunoglobulin (IVIG), and/or other compounds containing, or that can be metabolized into sialic acid, within 6 months prior to enrollment as reported by subject at the time of screening.
• •11. Subject has received stem cell therapy or gene therapy within 1 year prior to screening.
• •12. Subject has hypersensitivity to ManNAc or erythritol or in the judgment of the investigator, has a condition that places the subject at increased risk for adverse effects.
• •13. The presence of persistent diarrhea or malabsorption that could interfere with the subject's ability to absorb drugs or to tolerate ManNAc therapy.