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Expert opinion CF patient-derived tissue models are being explored to determine donor-specific response to current approved and future novel CFTR modulators for F508del and other rare mutations. The discovery and validation of biomarkers of CFTR modulation will complement these studies in the long term and in real-life world.Introduction As we have just stepped into a new decade of hopes, the mountain of knowledge learned from multiple myeloma (MM) remains unmatched among cancers. In the last decade alone, this rapid-sequence learning curve has led to regulatory approvals of eight drugs with mechanisms of actions representing five different areas of cell biology some of which made to the frontline setting, sparking debates about how to best sequence them in the treatment continuum of induction, consolidation, and maintenance and gained momentum with the realization of the implications of an effective upfront therapeutic approach with potential impact on survival.Areas covered This review was written with an intent to introduce the reader to the current treatment approach of a newly diagnosed myeloma patient and acquaint with promising targets and mechanistic strategies. Medline and clinicaltrials.gov databases (2000-2020) and relevant meetings (ASH, ASCO, EHA, ESMO, IMW) reports were queried and guidelines (IMWG) were reviewed to distill to expert opinion in an inundating field.Expert opinion Future holds promise with new targets on the horizon. https://www.selleckchem.com/products/monastrol.html It is likely that the new age of myeloma will belong to quadruplets with the addition of acellular or cellular biologics to first-generation novel agents, leading to new paradigms.Objective The aim of this meta-analysis was to examine the association of ApoE polymorphism with the risk of developing PE.Methods A comprehensive search was carried out through PubMed, Scopus, and Embase. The ORs with corresponding 95% CIs were extracted. Fixed model was used for meta-analysis and in case of existing heterogeneity a random-effects model was applied.Results Association of ApoE polymorphism with the risk of developing PE was not statistically significant (OR = 0.86, 95% CI 0.67-1.11; OR = 0.92, 95%CI 0.73-1.15, respectively for ε2 and ε4).Conclusion ApoE polymorphism might not be associated with the risk of PE.Introduction Fatigue and apathy are two key non-motor symptoms in Parkinson's disease (PD), with documented negative impact on Quality of life (QoL) and a frequent burden for caregivers.Areas covered In this invited review, researchers from the Parkinson Foundation Centre of Excellence in non-motor research at King's College Hospital and King's College London comment on the latest pathophysiology, clinical phenomenology, the most frequently used scales for fatigue and apathy in PD with a focus on available therapeutic strategies.Expert opinion The identification of fatigue and apathy in PD is mainly hampered by the lack of a clear consensus on these subjective symptoms. The pathophysiological processes remain unclear, and the large variation in prevalence is likely due to the heterogeneous PD populations and the lack of an enriched cohort of people with fatigue and/or apathy as main symptoms. Treatment strategies, and especially level 1 evidence for specific treatments for fatigue and apathy in PD, remain scarce. The best evidence to date is doxepin, rasagiline and levodopa infusion therapy (for fatigue), and rivastigmine (for apathy). Further efforts should be made to properly identify these two major symptoms in PD, to correctly detect those who may benefit most from tailored personalized interventions.During the past decades, converging evidence from clinical, neuroimaging and neuroanatomical studies has demonstrated the key role of the cerebellum in the processing of non-motor aspects of language. Although more is known about the way in which the cerebellum participates in the mechanisms involved in written language, there is ambiguous information on its role in other aspects of language, such as in non-motor aspects of spoken language. Thus, to contribute additional insight into this important issue, in the present work, we review several original scientific papers focusing on the most frequent non-motor spoken language impairments evidenced in patients affected by cerebellar pathology, namely, verbal working memory, grammar processing and verbal fluency impairments. Starting from the collected data, we provide a common interpretation of the spoken language disorders in cerebellar patients, suggesting that sequential processing could be the main mechanism by which the cerebellum participates in these abilities. Indeed, according to the cerebellar sequential theory, spoken language impairments could be due to altered cerebellar function to supervise, synchronize and coordinate the activity of different functional modules, affecting the correct optimization of linguistic processing.Introduction Preterm birth is the leading cause of neonatal morbidity and mortality globally and poses a substantial economic burden. Consequently, there is a need for the identification of therapeutic targets and novel experimental drugs for the inhibition of preterm labour to improve neonatal outcomes.Areas covered The authors review the pathophysiology of labour and the inflammatory pathways underpinning it. The interruption of these pathways forms the basis of therapeutic targets to inhibit preterm labour. Current drugs available for the treatment of preterm labour are reviewed, followed by experimental drugs; this includes toll-like receptor 4 (TLR-4) antagonists, cytokine suppressive anti-inflammatory drugs (CSAIDs), N-acetyl cysteine (NAC), Sulfasalazine (SSZ), tumour necrosis factor alpha (TNF-α) antagonists, interleukin-1 receptor (IL-1) inhibitors, omega-3 polyunsaturated fatty acids and lipid metabolites, and the polyphenols.Expert Opinion The drive to develop therapeutic strategies for the prevention of preterm labour is constantly evolving, hence significant opportunities for the improvement of survival and neurodevelopmental outcomes for babies born preterm and the reduction of healthcare burden now exist. Numerous therapeutics are being explored for their potential in preterm birth prevention, and it is likely that over the next decade there will be a new treatment option that targets the pathological inflammatory processes involved in preterm labour.
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