A Study to Assess Efficacy and Safety of RT001 in Subjects With Infantile Neuroaxonal Dystrophy

This is a single arm open-label study with a structured observation of INAD patients treated
with RT001. Enrolled subjects will undergo observation and testing to determine the effect of
RT001 treatment. Fifteen eligible subjects will be treated with RT001 for long-term
evaluation of efficacy, safety, tolerability, and pharmacokinetics.

Inclusion Criteria:

1. Male or female 18 months to 10 years of age

2. Medical history consistent with the symptoms of classic INAD (onset of symptoms
between the ages of 6 months and 3 years)

3. Homozygous for PLA2G6 deficiency (variant alleles may be mixed heterozygotes)

4. Must have impairment in at least 2 of the assessed categories at baseline

5. Signed informed consent form (ICF) prior to entry into the study

6. Able to provide the necessary blood samples

Exclusion Criteria:

1. Received treatment with other experimental therapies within the last 30 days prior to
the first dose

2. Requiring mechanical ventilation, other than positive air pressure support primarily
for mitigation of sleep apnea.

3. Have a life expectancy of less than one year

4. Diagnosis of atypical NAD (ANAD)

5. Unwilling or unable to comply with the requirements of this protocol, including the
presence of any condition (physical, mental, or social) that is likely to affect the
subject's ability to return for visits as scheduled