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Edaravone for the Treatment of Motor Neurone Disease: A Critical Review of Approved and Alternative Formulations against a Proposed Quality Target Product Profile

Edaravone is one of two main drugs for treating motor neurone disease (MND). This review proposes a specific quality target product profile (QTPP) for edaravone following an appraisal of the issues accounting for the poor clinical uptake of the approved IV and oral liquid edaravone formulations. This is followed by a review of the alternative oral formulations of edaravone described in the published patent and journal literature against the QTPP.

Hospitalizations Following Complex Hip Surgery in Children with Intellectual Disability: A Self-Controlled Case Series Analysis

To evaluate the associations between complex hip surgery and subsequent hospitalizations in children with intellectual disability, including a subset of children with cerebral palsy.

Optimising detection of thrombosis in paediatric Staphylococcus aureus bacteraemia: A prospective interventional sub-study protocol

Staphylococcus aureus bacteraemia (SAB) is the most common cause of sepsis, contributing to paediatric intensive care unit admission in Australia and New Zealand. While deep venous thrombosis (DVT) has been reported in children with invasive S. aureus infections, the actual frequency and possible effects of thrombosis on disease severity and outcome in paediatric SAB remain unknown. Moreover, guidance regarding imaging for paediatric SAB management are poorly defined. 

Net Promoter Score Model for Evaluating Paediatric Medicine Acceptability: Validation and Feasibility Study

Medicine acceptability is crucial for paediatric drug development, yet its assessment remains challenging due to the multifaceted nature of sensory attributes like taste, smell, and mouthfeel. Traditional methods of acceptability evaluation often involve complex questionnaires and lack standardisation, leading to difficulties in a comparative analysis across studies.

Antecedent and persistent symptoms in COVID-19 and other respiratory illnesses: Insights from prospectively collected data in the BRACE trial

Some individuals have a persistence of symptoms following both COVID-19 (post-acute COVID-19 syndrome; PACS) and other viral infections. This study used prospectively collected data from an international trial to compare symptoms following COVID-19 and non-COVID-19 respiratory illness, to identify factors associated with the risk of PACS, and to explore symptom patterns before and after COVID-19 and non-COVID-19 respiratory illnesses.

The effect and control of malaria in pregnancy and lactating women in the Asia-Pacific region

Half of all pregnancies at risk of malaria worldwide occur in the Asia-Pacific region, where Plasmodium falciparum and Plasmodium vivax co-exist. Despite substantial reductions in transmission, malaria remains an important cause of adverse health outcomes for mothers and offspring, including pre-eclampsia. Malaria transmission is heterogeneous, and infections are commonly subpatent and asymptomatic.

Subcutaneous infusion of high-dose benzathine penicillin G is safe, tolerable, and suitable for less-frequent dosing for rheumatic heart disease secondary prophylaxis: a phase 1 open-label population pharmacokinetic study

Since 1955, the recommended strategy for rheumatic heart disease secondary prophylaxis has been benzathine penicillin G injections administered intramuscularly every 4 weeks. Due to dosing frequency, pain, and programmatic challenges, adherence is suboptimal. It has previously been demonstrated that BPG delivered subcutaneously at a standard dose is safe and tolerable and has favorable pharmacokinetics, setting the scene for improved regimens with less frequent administration.

Population pharmacokinetics of penicillin G: insights into increased clearance at low concentrations to guide development of improved long-acting formulations for syphilis

Although benzylpenicillin (penicillin G) is listed by the World Health Organization as an Essential Medicine, dose optimization is a persistent challenge, especially for long-acting intramuscular formulations. Maintaining sustained antibiotic exposure at target concentrations is crucial for secondary chemoprophylaxis of rheumatic heart disease and treatment of syphilis. 

Severe adverse reactions to benzathine penicillin G in rheumatic heart disease: A systematic review and meta-analysis

Fear of severe adverse reaction (SAR) and reluctance of health care providers to administer intramuscular injections are major contributing factors to poor adherence of benzathine penicillin G (BPG) in the management of rheumatic heart disease (RHD). However, data on the risk of SARs following BPG injections for RHD are relatively limited and inconclusive. Our systematic review and meta-analysis aimed to evaluate the incidence of SARs associated with BPG injections used for secondary prophylaxis of RHD. 

Factors influencing adult and adolescent completion of treatment for late syphilis: a mixed methods systematic review

To identify factors influencing the completion of a three-dose course of weekly intramuscular benzathine penicillin G injections by adults and adolescents with syphilis of unknown duration or late syphilis.