Participate in Research at CU Anschutz

Child

- Male and female patients < 2 years of age at the time of informed consent - Genetically confirmed SMA diagnosis - Has received onasemnogene abeparvovec for SMA no less than 3 months prior to enrollment - In the opinion of the investigator, has demonstrated a plateau or decline in function post-gene therapy- 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 prior to enrollment - In the opinion of the investigator, has demonstrated a plateau or decline in function post-gene therapy (with a duration of 6 months or less) - 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