High Dose Ascorbic Acid Treatment of CMT1A

Inclusion Criteria:

• The subject has CMT1A, defined by the duplication on chromosome 17p11.2 performed byeither Pulse Field Gel Electrophoresis or Fluorescence In Situ Hybridization (FISH) bya CLIA certified laboratory, OR the subject has a first or second degree relative witha documented duplication performed by the above methods AND the subject has uniformmotor conduction slowing of the median or ulnar nerve between 16 and 30 m/s.

• The subject is between 13 and 70 years of age.

• The subject, if 18 years or older, has signed the Informed Consent Form and agrees tofollow the stipulations of the protocol.

• If the subject is less than 18, his or her parents or guardians have signed theInformed Consent Form and agree to follow the stipulations of the protocol. Thesubject has also signed a written assent form.

Exclusion Criteria:

• A known neuropathy from another source (For example, diabetes, drug induced, alcohol,etc.)

• The subject has ever received Vincristine.

• The subject has a known allergy to ascorbic acid.

• The subject has ever had kidney stones.

• The subject has a known history of G6PD deficit.

• The subject has a history of hemochromatosis.

• The subject suffers from a serious illness or medical condition that is not stabilizedor that could require hospitalization.

• The subject has a high ascorbic acid level at screening.

• The subject is pregnant or nursing.

• The subject, in the opinion of the investigator, is unlikely to comply with the studyprotocol or is unsuitable for any other reason.

• The subject participates to another clinical trial or is still within a washout periodof a previous clinical trial.

• The subject is taking neurotoxic medications.