Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
Zarife Sahenk
This study will assess the serum NT-3 levels in those with a peripheral neuropathy diagnosis which will help to determine the therapeutic levels of NT-3 in subjects that will receive AAV1.NT-3 gene therapy in a future study. This study also aims to define the natural history of any type of Charcot-Marie Tooth Disease (CMT), better define the rate of disease progression and skeletal muscle involvement. Another aim is to generate a registry of well-characterized CMT patients who may be candidates for future trials.
Age
7–any
Sex
ALL
Healthy volunteers
Not accepted
You may be eligible if
Males or females from 7 years of age or older
Diagnosis of acquired peripheral neuropathy (PN) such as due to diabetes, chemo-induced, autoimmune chronic inflammatory demyelinating polyneuropathy (CIDP) established by a report of electrical studies or with a known genetic CMT diagnosis. Definitive diagnosis of PN is established with a report of electrical studies, EMG/nerve conduction studies.2
Perform assessments to the best of their ability with reliable results as deemed by the evaluator.
Ability to attend scheduled appointments
Ability to provide informed consent (or assent for ages 9-18)
You may not be if
Current pregnancy per medical history
Has a medical condition or extenuating circumstances that, in the opinion of the investigator, might compromise the subject's wellbeing, safety, or clinical interpretability
