Methods | Randomised controlled double‐blind trial. | |
Participants | 8 patients with CMT1A carrying the duplication on chromosome 17p11.2 harbouring the PMP22 gene. | |
Interventions | One group 50 µg/ kg 3x/w recombinant human NT‐3 subcutaneously. Control group placebo injections for 6 months. | |
Outcomes | Nerve biopsies, Neuropathy impairment scale, pegboard test, density of myelinated fibers and Schwann cells in sural nerve biopsies. | |
Notes | No drop outs | |
Risk of bias | ||
Bias | Authors' judgement | Support for judgement |
Allocation concealment? | Low risk | A ‐ Adequate |