We report the recruitment activities and outcomes of a multi-disease neuromuscular patient registry in Canada. The Canadian Neuromuscular Disease Registry (CNDR) registers individuals across Canada with a confirmed diagnosis of a neuromuscular disease. Diagnosis and contact information are collected across all diseases and detailed prospective data is collected for 5 specific diseases: Amyotrophic Lateral Sclerosis (ALS), Duchenne Muscular Dystrophy (DMD), Myotonic Dystrophy (DM), Limb Girdle Muscular Dystrophy (LGMD), and Spinal Muscular Atrophy (SMA). Since 2010, the CNDR has registered 4306 patients (1154 pediatric and 3148 adult) with 91 different neuromuscular diagnoses and has facilitated 125 projects (73 academic, 3 not-for-profit, 3 government, and 46 commercial) using registry data. In conclusion, the CNDR is an effective and productive pan-neuromuscular registry that has successfully facilitated a substantial number of studies over the past 10 years.

Registry SES Socioeconomic status AIM To determine whether inequities in health outcomes for Indigenous Canadians are also present in cerebral palsy (CP) by comparing CP profiles between Indigenous and non-Indigenous children. METHOD Using the Canadian Cerebral Palsy Registry, we conducted a cross-sectional study. CP motor subtype, gross motor severity, comorbidities, perinatal adversity, preterm birth, and parental education were compared between 94 Indigenous (53 males, 41 females) and 1555 non-Indigenous (891 males, 664 females) children (all >5y). Multivariate analysis was done to analyze adverse CP factors, defined as CP gross motor severity and comorbidities. CP etiologies, either prenatal/perinatal or postnatal, were also compared. RESULTS Indigenous children with CP have higher odds of having low parental education (odds ratio [OR] 6.15, 95% confidence interval [CI] 3.36-11.3) and comorbidities (OR 4.46, 95% CI 1.62-12.3), especially cognitive (OR 4.52, 95% CI 2.27-9.05), communication (OR 2.66, 95% CI 1.54-4.61), and feeding (OR 2.25, 95% CI 1.33-3.83) impairment. Indigenous children also have higher CP gross motor severity (p=0.03). Indigenous children are also more likely to have nonaccidental head injury (n=4; OR 8.18, 95% CI 1.86-36.0) as the cause of their postnatal CP. INTERPRETATION Indigenous populations have worse health outcomes as a result of intergenerational impacts of colonization. Our study shows that Indigenous children with CP have increased comorbidities and higher CP gross motor severity, reinforcing the need for a multidisciplinary approach to management. Furthermore, targeted prevention programs against preventable causes of CP, such as non-accidental head injury, may be beneficial.

Background: 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. Methods: 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. Results: 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. Conclusion: 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.

Pain is a significant health concern for children living with cerebral palsy (CP). There are no population‐level or large‐scale multi‐national datasets using common measures characterizing pain experience and interference (ie, pain burden) and management practices for children with CP. The aim of the CPPain survey is to generate a comprehensive understanding of pain burden and current management of pain to change clinical practice in CP. The CPPain survey is a comprehensive cross‐sectional study. Researchers plan to recruit approximately 1400 children with CP (primary participants) across several countries over 6‐12 months using multimodal recruitment strategies. Data will be collected from parents or guardians of children with CP (0‐17 years) and from children with CP (8‐17 years) who are able to self‐report. Siblings (12‐17 years) will be invited to participate as controls. The CPPain survey consists of previously validated and study‐specific questionnaires addressing demographic and diagnostic information, pain experience, pain management, pain interference, pain coping, activity and participation in everyday life, nutritional status, mental health, health‐related quality of life, and the effect of the COVID‐19 pandemic on pain and access to pain care. The survey will be distributed primarily online. Data will be analyzed using appropriate statistical methods for comparing groups. Stratification will be used to investigate subgroups, and analyses will be adjusted for appropriate sociodemographic variables. The Norwegian Regional Committee for Medical and Health Research Ethics and the Research Ethics Board at the University of Minnesota in USA have approved the study. Ethics approval in Canada, Sweden, and Finland is pending. In addition to dissemination through peer‐reviewed journals and conferences, findings will be communicated through the CPPain Web site (http://www.sthf.no/cppain), Web sites directed toward users or clinicians, social media, special interest groups, stakeholder engagement activities, articles in user organization journals, and presentations in public media.