Age
Child to Adult
Eligibility
- Male and female patients < 2 years of age at the time of informed consent
- Genetically confirmed diagnosis of SMA
- s received onasemnogene abeparvovec for SMA no less than 3 months, but not more than 7 months, prior to enrollment
- Has, in the opinion of the investigator, not experienced clinically significant decline
in function from the time of onasemnogene abeparvovec administration- Provision of Informed Consent Form by a legally authorized representative
- Male and female patients < 2 years of age at the time of informed consent
- Confirmed diagnosis of 5q-autosomal recessive SMA, including genetic confirmation
of homozygous deletion or compound heterozygosity predictive of loss of function of
the SMN1 gene
- Confirmed presence of two SMN2 gene copies as documented through laboratory
testing
- Administration of onasemnogene abeparvovec pre-symptomatically or
post-symptomatically
- Has received onasemnogene abeparvovec for SMA no less than 3 months, but not
more than 7 months, prior to enrollment
- Has, in the opinion of the investigator, not experienced clinically significant decline
in function from the time of onasemnogene abeparvovec administration
- Receiving adequate nutrition and hydration (with or without gastrostomy) at the time
of screening, in the opinion of the investigator
- Adequately recovered from any acute illness at the time of screening and
considered well-enough to participate in the opinion of the investigator
- Medical care meets, in the opinion of the investigator, locally accepted standard of
care
- Expected to be able to safely travel to the study site for the whole duration of the
study and according to the frequency of required study visits, in the opinion of the
investigator
- Have a stable home situation with a consistent caregiver
- Able to complete all study procedures, measurements, and visits, and the parent or
caregiver of the patient, in the opinion of the investigator, has adequately supportive
psychosocial circumstances
- Parent or caregiver of patient willing to consider nasogastric, naso-jejunal, or
gastrostomy tube placement during the study to maintain safe hydration, nutrition,
and treatment delivery, as recommended by the investigator
- Parent or caregiver of patient willing to consider the use of non-invasive ventilation
during the study, as recommended by the investigator
- Ability of patient and caregivers to comply with the study protocol
