Clinical drug investigation
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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.
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Review Meta Analysis
Efficacy and Safety of Dexmedetomidine as an Adjuvant in Epidural Analgesia and Anesthesia: A Systematic Review and Meta-analysis of Randomized Controlled Trials.
Several clinical trials have examined and indicated the usefulness of epidural dexmedetomidine therapy. However, there has been no systematic analysis of the findings of these trials to date. We undertook this systematic review and meta-analysis to investigate the efficacy and safety of epidural dexmedetomidine adjunctive therapy in different surgical procedures. ⋯ This systematic review and meta-analysis demonstrates that dexmedetomidine as an adjuvant in epidural procedures is generally safe and well tolerated. Furthermore, dexmedetomidine acted synergistically and provided an improved sedation and analgesic profile.
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Review Meta Analysis
Efficacy and Safety of Dexmedetomidine as an Adjuvant in Epidural Analgesia and Anesthesia: A Systematic Review and Meta-analysis of Randomized Controlled Trials.
Several clinical trials have examined and indicated the usefulness of epidural dexmedetomidine therapy. However, there has been no systematic analysis of the findings of these trials to date. We undertook this systematic review and meta-analysis to investigate the efficacy and safety of epidural dexmedetomidine adjunctive therapy in different surgical procedures. ⋯ This systematic review and meta-analysis demonstrates that dexmedetomidine as an adjuvant in epidural procedures is generally safe and well tolerated. Furthermore, dexmedetomidine acted synergistically and provided an improved sedation and analgesic profile.
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Methoxyflurane (Penthrox®) is a halogenated ether first used clinically as a volatile inhalational anaesthetic. It has been used as an analgesic in Australia and New Zealand for the past 30 years. In the UK and Europe, methoxyflurane has been approved for the emergency relief of moderate to severe trauma pain in conscious adult patients. ⋯ Although methoxyflurane is known to be potentially nephrotoxic at anaesthetic doses, the much lower doses used for pain relief were not associated with nephrotoxicity or an increased risk of renal disease. Inhaled methoxyflurane may offer advantages over other analgesics administered via the intravenous, intramuscular or intranasal routes in terms of its non-invasive self-administration, ease of use and/or rapid onset of action. As such, it is a useful additional treatment option for the management of trauma pain in the pre-hospital or emergency department setting.
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Review Meta Analysis
Umeclidinium Plus Vilanterol Versus Placebo, Umeclidinium, or Vilanterol Monotherapies for Chronic Obstructive Pulmonary Disease: A Meta-Analysis of Randomized Controlled Trials.
The role of umeclidinium plus vilanterol as a combination therapy for chronic obstructive pulmonary disease (COPD) has not yet been clearly defined. ⋯ Compared with the other three groups, i.e. placebo, umeclidinium and vilanterol, umeclidinium plus vilanterol improves lung function and quality of life in patients with COPD, reduces the use of albuterol, and does not increase the incidence of adverse events and serious adverse events.