A Study of Risdiplam (RO7034067) in Adult and Pediatric Participants With Spinal Muscular Atrophy

Inclusion Criteria:

- Confirmed diagnosis of 5q-autosomal recessive SMA

- Previous enrollment in Study BP29420 (Moonfish) with the splicing modifier RO6885247
or previous treatment with any of the following: 1.) Nusinersen (defined as having
received >= 4 doses of nusinersen, provided that the last dose was received >= 90 days
prior to screening) or 2.) Olesoxime (provided that the last dose was received <= 12
months and >= 90 days prior to screening)

- Adequately recovered from any acute illness at the time of screening and considered
well enough to participate in the opinion of the Investigator

- For women of childbearing potential: negative blood pregnancy test at screening,
agreement to remain abstinent (refrain from heterosexual intercourse) or use
contraceptive measures, and agreement to refrain from donating eggs

- For men: agreement to remain abstinent (refrain from heterosexual intercourse) or use
contraceptive measures and agreement to refrain from donating sperm

- For patients aged 2 years or younger at screening: 1.) Parent or caregiver of patient
is willing to consider nasogastric, naso-jejunal or gastrostomy tube placement, as
recommended by the Investigator, during the study (if not already in place at the time
of screening) to maintain safe hydration, nutrition and treatment delivery. 2.) Parent
or caregiver of patient is willing to consider the use of non-invasive ventilation, as
recommended by the Investigator during the study (if not already in place at the time
of screening).

Exclusion Criteria:

- Inability to meet study requirements

- Concomitant participation in any investigational drug or device study

- Previous participation in any investigational drug or device study, with the exception
of studies with olesoxime or nusinersen, within 90 days prior to screening, or 5
half-lives of the drug, whichever is longer

- Any history of gene or cell therapy

- Unstable gastrointestinal, renal, hepatic, endocrine, or cardiovascular system
diseases as considered to be clinically significant by the Investigator

- Inadequate venous or capillary blood access for the study procedures, in the opinion
of the Investigator

- For patients aged < 2 years, hospitalization for a pulmonary event within 2 months
prior to screening and pulmonary function not fully recovered at the time of screening

- Lactating women

- Suspicion of regular consumption of drugs of abuse

- For adults and adolescents only, positive urine test for drugs of abuse or alcohol at
screening or Day -1 visit

- Presence of clinically significant electrocardiogram (ECG) abnormalities before study
drug administration from average of triplicate measurement or cardiovascular disease

- For patients aged > 6 years, significant risk for suicidal behavior, in the opinion of
the Investigator as assessed by the C-SSRS

- Any major illness within one month before the screening examination or any febrile
illness within one week prior to screening and up to first dose administration

- Recently initiated treatment for spinal muscular atrophy (within 6 weeks prior to
enrollment) with oral salbutamol or another beta 2-adrenergic agonist taken orally

- Any prior use of chloroquine, hydroxychloroquine, retigabin, vigabatrin or
thioridazine, is not allowed

- Ascertained or presumptive hypersensitivity (e.g., anaphylactic reaction) to RO7034067
or to the constituents of its formulation - Concomitant disease or condition that
could interfere with, or treatment of which might interfere with, the conduct of the
study, or that would, in the opinion of the Investigator, pose an unacceptable risk to
the participant in this study

- Recent history (less than one year) of ophthalmological diseases that would interfere
with the conduct of the study as assessed by an ophthalmologist. Participants in whom
Organic Cation Transporter or Spectral Domain-Optical Coherence Tomography (SD-OCT)
measurement of sufficient quality cannot be obtained at screening will not be enrolled