Table 1 Inclusion and exclusion criteria

Age between 4–65 at enrollment

Any other illness that would interfere with the ability to undergo safe testing or would interfere with interpretation of the results in the opinion of the site investigator

Clinically affected (defined as weakness on bedside evaluation in either a limb-girdle pattern or in a distal extremity)

Genetically or functionally confirmed mutation in ANO5, CAPN3, DYSF, SGCA-G, or DNAJB6

History of

• Bleeding disorder

• Platelet count < 50,000

• Current use of an anticoagulant

Willing and able to give informed consent and follow all study procedures and requirements

Positive pregnancy test at any time point during the trial