Spinal Muscular Atrophy
Conditions
Interventions
None listed
Sponsors
Eligibility
Inclusion criteria
Inclusion criteria: 1)Patients with clinically diagnosed SMA, aged 18 years and older at the time of enrollment. 2)SMA patients with genetically confirmed loss of the SMN1 gene as well as retention of at least one copy of the SMN2 gene. 3)SMA patients who are available for follow-up visits. 4)SMA patients who can provide written consent for participation in the study.
Exclusion criteria
Exclusion criteria: 1)SMA patients who have psychiatric disorder. 2)SMA patients who are judged as unsuitable for the study by the investigator.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Collecting longitudinal data of Motor function and QOL/PRO | — |
Countries
Japan
Contacts
Mebix, Inc. Research Promotion Group