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. 2024 Nov 12;22:eAE1133. doi: 10.31744/einstein_journal/2024AE1133

Table 1. Study enrolment criteria.

Inclusion criteria
1. Genetically confirmed 5q-SMA patients at all ages
2. Consent to participate in the study, expressed by the patient or responsible or legal guardian of the pediatric patient/ responsible or legal guardian of the patient with cognitive impairment of understanding the registration protocol
Exclusion criteria
1. Patients without a genetic diagnosis confirming 5q-SMA
2. Other types of SMA (non 5q-SMA)
3. Patients who do not accept to participate in the observational study
4. Patients without the legal capacity, who are unable to understand the nature, significance, and consequences of participating in the registry, or, in such cases, without a legal or responsible guardian