Delivery of therapeutics to the brain and spinal cord remains a challenge for neurodegenerative diseases, such as spinal muscular atrophy (SMA) and amyotrophic lateral sclerosis (ALS). The blood-brain-barrier (BBB) hampers delivery of therapeutics to neurons, glia, and surrounding cell types of the central nervous system (CNS) that may be involved in disease progression. Here, we describe an intravenous injection of adeno-associated-virus-9 (AAV-9) in mouse that efficiently targets brain, dorsal root ganglia and spinal cord motor neurons in neonatal animals and astrocytes in adult mouse brain and spinal cords, offering a new therapeutic delivery approach to deliver genes to widespread regions within the CNS.

There are few large studies investigating rates of infections in patients with autoimmune neuromuscular disorders and the relationship to immunomodulation. This study not only demonstrates the remarkably similar infection rates across the 3 diseases studied, but also shows their relationship to commonly used immunotherapies. Muscle Nerve 57: 927-931, 2018.

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Transition from pediatric to adult health care is a time of high risk for adolescents and young adults (AYAs) with epilepsy. Our aims are to examine patient, caregiver, and provider perceptions of transition readiness and the influence of patient-provider alliance in the context of transition readiness. Our cross-sectional study included 82 AYAs with epilepsy prior to transition. Patients, caregivers, and providers completed questionnaires (e.g., transition readiness and working alliance). Statistical analyses included independent samples and paired t-tests. Participants were 17.3 ± 2.8 years on average, 54% were females, 84% were White (non-Hispanic), and 38% had generalized epilepsy. Caregivers reported significantly higher transition readiness than their AYAs (t (72) = −10.6, p < 0.001). AYAs and providers reported similar alliance scores. Providers who felt patients were ready to transition had higher patient-reported transition readiness and provider-reported alliance scores. These data suggest that patients and providers are well aligned in the transition process, and providers appropriately perceive key areas necessary for transition. Caregivers and patients had discrepant perceptions of transition readiness, highlighting the importance of assessing both unique transition perspectives. Dedicated transition programs are likely to be beneficial in improving transition readiness and increase alignment across patients, caregivers, and providers.