A Study of RO6885247 in Adult and Pediatric Patients With Spinal Muscular Atrophy (MOONFISH)

Inclusion Criteria:

- Males and females, aged 2 to 55 years inclusive or below 7 months inclusive

- Confirmed diagnosis of 5q-autosomal recessive SMA (Types 1 to 3), for patients aged 7
months or below clinical symptoms attributable to type 1 SMA and 2 SMN2 copies

- Able and willing to provide informed consent and to comply with the study protocol.
Alternatively, a legally authorized representative must be able to consent for the
patient and assent must be given by the subject wherever possible.

- Female patients of childbearing potential and male patients with a female partner of
childbearing potential must agree with the required contraceptive methods as defined
per protocol.

- For patients aged 7 months or below, Gestational age of 37 to 42 weeks and not
considered small for gestational age at birth

Exclusion Criteria:

- Concomitant or previous participation in any investigational drug or device study
within 90 days prior to screening

- Concomitant or previous participation in a SMN2-targeting antisense oligonucleotide
study within 12 months prior to screening

- Concomitant or previous participation at any time in a gene therapy study

- For patients aged 2-55 years, hospitalization for pulmonary event within the last 2
months or planned at the time of screening

- Surgery for scoliosis in the last 6 months from screening or planned within 6 months
from screening

- Unstable gastrointestinal, renal, hepatic, endocrine or cardiovascular system disease

- Clinically relevant ECG abnormalities at screening or baseline; personal or family
history (first degree relatives) of congenital long QT syndrome

- Clinically significant abnormalities in laboratory test results at screening

- Any concomitant disease or condition that could interfere with the conduct of the
study, or pose an unacceptable risk to the subject in this study

- Use of prohibited medications as per protocol within 90 days prior to randomization.
Patients who are on inhaled corticosteroids, administered either through a nebulizer
or an inhaler, are allowed.

- Recently initiated treatment (within <6 months prior to randomization) with oral
salbutamol or another beta2-adrenergic agonist taken orally is not allowed. Patients
who have been on oral salbutamol (or another beta2-adrenergic agonist) for at least 6
months before randomization are allowed. Use of inhaled beta2-adrenergic agonists is
allowed.

- For patients aged 7 months or below, patients requiring invasive ventilation or
tracheostomy, presence of non-SMA related morbidities