Journal of pediatric orthopedics
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Many patients with spinal muscular atrophy (SMA) who might benefit from intrathecal antisense oligonucleotide (nusinersen) therapy have scoliosis or spinal fusion that precludes safe drug delivery. To circumvent spinal pathology, we designed a novel subcutaneous intrathecal catheter (SIC) system by connecting an intrathecal catheter to an implantable infusion port. ⋯ Preliminary observations reveal the SIC to be relatively safe and well tolerated in SMA patients with advanced disease and spinal fusion. The SIC warrants further study and, if proven effective in larger trials of longer duration, could double the number of patients able to receive nusinersen worldwide while reducing administration costs 5- to 10-fold.
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To report a single-center surgical experience treating humeral deformity and fractures in children with osteogenesis imperfecta (OI) using the Fassier-Duval (FD) intramedullary elongating rods. ⋯ Level IV-retrospective case series.
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The evidence-based approach to guide clinical practice has gained great importance in the medical field. High-quality evidence is of paramount importance to inform clinical decision-making and optimize patient outcomes. The generation of high-quality evidence relies on sound methodology and study design to facilitate appropriate interpretation and subsequent application of the clinical findings. The purpose of this review is to objectively critique the methodological design and clinical findings of 2 pediatric orthopaedic studies on children with cerebral palsy (CP) to assess their potential to impact clinical practice. ⋯ Despite a positive result demonstrating an effect of botulinum toxin treatment on spastic equinus in CP, the long-term functional impact and optimal dose remains to be determined. In addition, the negative result in the second study demonstrates the need for a noninferiority trial design to appropriately demonstrate no difference between 2 treatment options.
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The objective of this study was to determine if adolescent and young adult patients undergoing hip arthroscopy for symptomatic femoroacetabular impingement (FAI) experience clinically meaningful improvements in functional outcome scores. ⋯ Level IV-therapeutic case series.
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The purpose of this study was to evaluate the type and severity of orthopaedic and associated injuries for snowmobile, All-terrain vehicles (ATV) and motorized dirtbike accidents in a pediatric patient population. ⋯ Pediatric snowmobile, ATV and dirtbike accidents result in severe orthopaedic and associated injuries with each vehicle demonstrating significantly different injury patterns. Injury prevention should focus on improved safety mechanisms, protective gear, safe areas for off-road vehicle use and strict laws with minimum age requirements LEVEL OF EVIDENCE:: Level IV.