An Open Label Study of Gene Therapy Product in Spinal Muscular Atrophy Patients
This is an interventional study to evaluate safety and efficacy of AAV-hSMN1 in spinal muscular atrophy patients.
- EligibilityCriteria: Inclusion Criteria:  Diagnosis of SMA based on gene mutation analysis with bi-allelic survival motor neuron (SMN1) mutations (deletion or point mutations). Patients or Parent(s)/legal guardian(s) willing and able to complete the informed consent process and comply with study procedures and visit schedule.  Exclusion Criteria:  Active viral infection (includes HIV or serology positive for hepatitis B or C). Use of invasive ventilatory support (tracheotomy with positive pressure) or pulse oximetry <95% saturation. Concomitant illness and any drug that in the opinion of the investigator creates unnecessary risks for gene transfer. Clinically significant abnormal laboratory values. Participation in a recent SMA treatment clinical trial that in the opinion of the PI creates unnecessary risks for gene transfer. Patient with signs of aspiration based on a swallowing test and unwilling to use an alternative method to oral feeding. - HealthyVolunteers: No - Gender: All - StdAgeList: Child, Adult, Older Adult