The journal of pediatric pharmacology and therapeutics : JPPT : the official journal of PPAG
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J Pediatr Pharmacol Ther · May 2019
ReviewNusinersen: A Novel Antisense Oligonucleotide for the Treatment of Spinal Muscular Atrophy.
Spinal muscular atrophy (SMA) encompasses a group of autosomal recessively inherited degenerative neuromuscular disorders. They range in severity from neonatal onset with rapidly progressive weakness and early mortality (SMA-1), to onset in infancy (SMA-2), to adolescent/adult onset with indolent clinical course (SMA-3/-4). SMA patients share mutations in the survival motor neuron (SMN) gene; variations in clinical phenotypes are attributable to copy numbers of the closely related SMN2 gene. ⋯ Treatment requires complex financial and logistics because of the very high drug cost, intrathecal administration, and medical fragility of the patients. Treatment implementation also engenders ethical considerations related to cost, insurance coverage, limited clinical data on groups of patients not in clinical trials, and questions of duration of treatment. Nusinersen has been integrated into the treatment of many SMA patients.
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J Pediatr Pharmacol Ther · May 2019
A Retrospective Review of Antipsychotic Medications Administered to Psychiatric Patients in a Tertiary Care Pediatric Emergency Department.
An increasing number of pediatric patients with psychiatric chief complaints present to emergency departments (EDs) nationwide. Many of these patients require treatment with antipsychotic medications to treat agitation. We sought to examine the use of antipsychotic medications in pediatric patients presenting to a tertiary care pediatric ED. ⋯ In this retrospective series, the majority of patients were treated with newer oral antipsychotics. Administration of multiple medications was associated with a significantly longer length of stay in the ED, as was parenteral administration of antipsychotics.
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J Pediatr Pharmacol Ther · May 2019
Case ReportsSugammadex for Reversal of Neuromuscular Blockade in a Patient With Renal Failure.
Residual neuromuscular blockade following the use of non-depolarizing neuromuscular blocking agents (NMBAs) can lead to postoperative respiratory complications, including oxygen desaturation, atelectasis, and pneumonia. Sugammadex rapidly reverses steroidal NMBAs by encapsulating them in a highly stable water-soluble complex. ⋯ We present a 19-year-old patient with renal failure who received sugammadex for reversal of neuromuscular blockade. The use of sugammadex in patients with renal dysfunction is discussed and safety concerns are reviewed.