Clinical drug investigation
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Review Meta Analysis
Interventions for Refractory Trigeminal Neuralgia: A Bayesian Mixed Treatment Comparison Network Meta-Analysis of Randomized Controlled Clinical Trials.
Patients with trigeminal neuralgia (TN) are often refractory to recommended first-line agents. Due to the absence of a systematic review, we undertook a network meta-analysis to assess various interventions that can be used to manage refractory TN. ⋯ We found that drug-related interventions that include sumatriptan, intranasal lidocaine, intravenous lidocaine, and botulinum toxin and combined continuous and pulsed radiofrequency thermocoagulation had significant effects in reducing pain in patients with refractory TN. However, the quality of evidence was graded as very low for all except botulinum toxin.
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Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disorder characterized by degeneration of spinal motor neurons and poses significant adverse outcome in affected population. Survival motor neuron 1 (SMN1) protein encoded by SMN1 gene located on 5q13 is critical for survival and functioning of motor neurons. Almost identical gene SMN2, present on the same chromosome, produces a small truncated protein (SMN2) because of skipping of exon 7 from translation due to translation silent C6U substitution in exon 7 of SMN2 pre-mRNA transcript. ⋯ Major evidence of its efficacy came from a sham controlled phase 3 clinical study ENDEAR. The study was stopped early based on significantly favorable results in interim analysis and all the patients were transitioned to receive nusinersen in an ongoing open-label, phase 3 study, SHINE, which will evaluate the long-term efficacy, safety and tolerability of the drug. Nusinersen is globally the first drug approved (by the US FDA) for treatment of SMA in children and adults.