The journal of pediatric pharmacology and therapeutics : JPPT : the official journal of PPAG
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J Pediatr Pharmacol Ther · Jan 2021
ReviewOnasemnogene Abeparvovec (AVXS-101) for the Treatment of Spinal Muscular Atrophy.
Spinal muscular atrophy (SMA) is a debilitating disorder characterized by degeneration of large motor neurons. It is a heterogeneous group of disorders caused by a homozygous deletion in the survival motor neuron (SMN) gene on chromosome 5, resulting in a SMN protein deficiency. Small amounts of SMN protein are also produced by the SMN2 gene, which that differs from SMN1 by a single nucleotide. ⋯ This one-time intravenous injection of this new gene therapy also bears a hefty price tag; however, it may be more cost effective in the long run versus the multiple intrathecal administrations needed with nusinersen. Drug access and use are hindered by drug cost, payer reimbursement issues, and lack of long-term data from clinical studies. Questions also remain regarding the safety and efficacy of repeated drug administration for patients with advanced disease.
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J Pediatr Pharmacol Ther · Jan 2021
Adolescents' Perspectives on Prescription Opioid Misuse and Medication Safety.
This study explored adolescents' perspectives on prescription opioids, opioid safety and misuse, and sources of opioid information. ⋯ Adolescents had some knowledge pertaining to prescription opioids, but they had misconceptions related to safety. Participants were aware of safety risks and negative consequences of misuse. Adolescents obtained medication information from various sources, including health care professionals, family and peers, and online sources. Educational efforts that target adolescents should provide opportunities for addressing misconceptions about safe and responsible use of medications.