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National Institutes of Health is Recruiting Patients For a ManNac Clinical Trial

Here is what many patients were waiting for and this is a very important opportunity to take part in this clinical trial that will take place at the NIH in Bethedsa, Maryland, U.S.A.  Please let me know if you need some one to assist you in getting in touch with the researchers due to language barriers or time zones.  Here is the information with the link at the end of the text.

An Open Label Phase 2 Study of DEX-M74 in Subjects With GNE Myopathy

- To evaluate the safety of 90 days of DEX-M74 given by mouth.
- Adults ages 18 60 diagnosed with GNE myopathy.
  • Eligible participants will be admitted to the NIH Clinical Center for the first visit and will stay 10 14 days. Participants will have:
  • Medical history and physical exam
  • Electrocardiogram to measure heart function
  • Blood and urine tests
  • Muscle strength tests
  • Magnetic resonance imaging (MRI) muscle scans. Participants will lie on a table that slides in and out of a metal tube that takes pictures
  • Questionnaires
  • Muscle biopsies. Samples of muscle will be taken, one each from the arm and leg.
  • The study drug as a liquid twice a day
  • Participants may wear a small activity monitor throughout the study. It can be worn on a waistband.
  • After discharge from the initial visit, participants will take the study drug at home. Participants will need to record if they miss any doses.
  • Visit 2 will be at 6 weeks for 1 2 days of medical evaluation. Blood samples will be drawn.
  • Visit 3 will be at the end of the study. Participants will stay in the NIH Clinical Center for 5 6 days for medical evaluations, muscle tests, and scans. Another muscle biopsy will be taken. Blood samples will be drawn.
  • Participants will be contacted by telephone or email about 4 times after leaving the clinic.


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