Publications by authors named "F J Ceravolo"
Objective: The impact of maternal stress on birth outcomes is well established in the scientific research. The sex ratio at birth (SRB), namely the ratio of male to female live births, shows significant alteration when mothers experience acute stress conditions, as proposed by the Trivers-Willard Hypothesis. We aimed to synthetize the literature on the relationship between two exogenous and catastrophic stressful events (natural disasters and epidemics) and SRB.
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- - Alpha-mannosidosis (AM) is a genetic disorder causing lysosomal enzyme deficiency, leading to the buildup of specific sugars; Velmanase alfa (VA) is the first enzyme replacement therapy targeting AM's non-neurological symptoms.
- - A study involving 33 patients on VA explored if genetic subgroups (G1, G2, G3) influenced antibody response (antidrug antibodies or ADAs) and related infusion reactions, finding that G1 and G2 were more likely to develop ADAs but most patients experienced mild or no infusion-related reactions.
- - Despite some patients developing ADAs, the overall treatment effects on serum oligosaccharides and clinical outcomes were similar across ADA-positive and ADA-negative
Article Synopsis
- - Alpha-mannosidosis (AM) is a rare genetic disorder caused by the deficiency of an enzyme, leading to the buildup of certain sugars, with symptoms that can be severe and often go undiagnosed until late childhood.
- - Velmanase alfa (VA) is the first enzyme replacement therapy approved in Europe for treating non-neurological symptoms of AM, with research suggesting earlier treatment could lead to better outcomes.
- - A phase 2 study involving five children under 6 years old found that long-term VA treatment was generally safe, well-tolerated, and resulted in improvements in health indicators, although the small sample size limits broader conclusions.
Article Synopsis
- - SPARKLE is a registry for alpha-mannosidosis aimed at gathering long-term safety and effectiveness data on velmanase alfa, the first enzyme replacement therapy for the disorder, particularly for non-neurologic symptoms in patients with mild to moderate cases.
- - The registry is a multicenter, multinational study, enrolling patients since 2020, and will track outcomes for up to 15 years, focusing on safety and effectiveness under routine clinical care.
- - Primary safety measures include monitoring adverse events and antibody development, while effectiveness is assessed through various health outcomes, providing insights into the disease's progression and natural history in treated and untreated patients.
Therapeutic plasma exchange, consisting of removing blood plasma and exchanging it with donated blood products, has been proposed for treating Alzheimer's disease (AD) to remove senescent or toxic factors. In preclinical studies, administration of plasma from young healthy mice to AD transgenic mice improved cognitive deficits without affecting brain amyloid plaques. Initial encouraging results have been collected in a double-blind, placebo-controlled study in nine AD patients receiving young plasma.
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