Spinal muscular atrophy (SMA) is a devastating rare disease that affects individuals regardless of ethnicity, gender, and age. The first-approved disease-modifying therapy for SMA, nusinursen, was approved by Health Canada, as well as by American and European regulatory agencies following positive clinical trial outcomes. The trials were conducted in a narrow pediatric population defined by age, severity, and genotype. Broad approval of therapy necessitates close follow-up of potential rare adverse events and effectiveness in the larger real-world population.

The Canadian Neuromuscular Disease Registry (CNDR) undertook an iterative multi-stakeholder process to expand the existing SMA dataset to capture items relevant to patient outcomes in a post-marketing environment. The CNDR SMA expanded registry is a longitudinal, prospective, observational study of patients with SMA in Canada designed to evaluate the safety and effectiveness of novel therapies and provide practical information unattainable in trials.

The consensus expanded dataset includes items that address therapy effectiveness and safety and is collected in a multicenter, prospective, observational study, including SMA patients regardless of therapeutic status. The expanded dataset is aligned with global datasets to facilitate collaboration. Additionally, consensus dataset development aimed to standardize appropriate outcome measures across the network and broader Canadian community. Prospective outcome studies, data use, and analyses are independent of the funding partner.

Prospective outcome data collected will provide results on safety and effectiveness in a post-therapy approval era. These data are essential to inform improvements in care and access to therapy for all SMA patients.

Whether the prevalence rates of common mental disorders can be compared across countries depends on the cultural validity of the diagnostic measures used.

To investigate the prevalence of Western and indigenously defined mental disorders among Vietnamese living in Vietnam and in Australia, comparing the data with an Australian-born sample.

Comparative analysis of three multistage population surveys, including samples drawn from a community living in the Mekong Delta region of Vietnam (n=3039), Vietnamese immigrants residing in New South Wales, Australia (n=1161), and an Australian-born population (n=7961). Western-defined mental disorders were assessed by the Composite International Diagnostic Interview (CIDI) 2.0 and included DSM–IV anxiety, mood and substance use disorders as well as the ICD–10 category of neurasthenia. The Vietnamese surveys also applied the indigenously based Phan Vietnamese Psychiatric Scale (PVPS). Functional impairment and service use were assessed.

The prevalence of CIDI mental disorders for Mekong Delta Vietnamese was 1.8% compared with 6.1% for Australian Vietnamese and 16.7% for Australians. Inclusion of PVPS mental disorders increased the prevalence rates to 8.8% for Mekong Delta Vietnamese and 11.7% for Australian Vietnamese. Concordance was moderate to good between the CIDI and the PVPS for Australian Vietnamese (area under the curve (AUC)=0.77) but low for Mekong Vietnamese (AUC=0.59). PVPS- and CIDI-defined mental disorders were associated with similar levels of functional impairment.

Cultural factors in the expression of mental distress may influence the prevalence rates of mental disorders reported across countries. The findings have implications for assessing mental health needs at an international level.