Publications by authors named "Fabio A Barroso"
Article Synopsis
- The study evaluates the efficacy of eplontersen, a new treatment targeting hereditary transthyretin amyloidosis (ATTRv), specifically in its polyneuropathy form, through a phase 3 trial known as NEURO-TTRansform.
- Conducted across 40 sites globally, the trial included 168 adults with specific eligibility criteria, comparing results from patients treated with eplontersen to historical data from placebo participants.
- Main outcomes measured improvements in serum transthyretin levels and quality of life scores, showing a significant reduction in transthyretin levels in eplontersen-treated patients compared to placebo, indicating potential effectiveness of the treatment.
Background And Objectives: To investigate CSF findings in relation to clinical and electrodiagnostic subtypes, severity, and outcome of Guillain-Barré syndrome (GBS) based on 1,500 patients in the International GBS Outcome Study.
Methods: Albuminocytologic dissociation (ACD) was defined as an increased protein level (>0.45 g/L) in the absence of elevated white cell count (<50 cells/μL).
Article Synopsis
- - Hereditary transthyretin (ATTRv) amyloidosis is a rare and severe disease caused by the buildup of transthyretin amyloid fibrils, affecting both men and women and leading to debilitating symptoms.
- - The NEURO-TTRansform study is a pivotal phase 3 clinical trial evaluating the drug Eplontersen for treating patients with ATTRv-PN, focusing on individuals who are ambulatory with specific disease stages and genetic variants.
- - A total of 168 patients were enrolled from 15 countries, with an average age of 52.8 years, predominantly male, and most had early disease onset, emphasizing the need for effective treatments in this population.
Background And Objectives: Infections play a key role in the development of Guillain-Barré syndrome (GBS) and have been associated with specific clinical features and disease severity. The clinical variation of GBS across geographical regions has been suggested to be related to differences in the distribution of preceding infections, but this has not been studied on a large scale.
Methods: We analyzed the first 1,000 patients included in the International GBS Outcome Study with available biosamples (n = 768) for the presence of a recent infection with , hepatitis E virus, , cytomegalovirus, and Epstein-Barr virus.
Article Synopsis
- - Autonomic dysfunction affects 40.4% of symptomatic patients with transthyretin amyloidosis (ATTR), occurring more frequently in those with the inherited variant (ATTRv) compared to the wild-type (ATTRwt).
- - Patients with ATTRv experience autonomic dysfunction symptoms much sooner (averaging 3.4 years) than those with ATTRwt (averaging 9.7 years).
- - While autonomic dysfunction significantly reduces quality of life in ATTRv patients, it does not appear to have the same impact in ATTRwt patients, as their quality of life scores are similar regardless of dysfunction presence.
Article Synopsis
- The study looked at a score called mEGOS that helps predict if people with a sickness called Guillain-Barré syndrome (GBS) will be able to walk on their own or not.
- Researchers used information from 1,500 patients from a big study to see if mEGOS worked well for people from different regions and made some improvements to it.
- The updated score showed good results in different areas, but some places had better or worse outcomes than expected, and they found that things like age and how weak someone’s limbs were were important for predicting problems.
Guillain-Barré syndrome is a heterogeneous disorder regarding the clinical presentation, electrophysiological subtype and outcome. Previous single country reports indicate that Guillain-Barré syndrome may differ among regions, but no systematic comparative studies have been conducted. Comparative studies are required to identify factors determining disease susceptibility, variation and prognosis, and to improve diagnostic criteria.
View Article and Find Full Text PDFBackground: Hereditary transthyretin (ATTRm) amyloidosis is a rare, progressive and fatal disease with a range of clinical manifestations.
Objective: This study comprehensively evaluates disease characteristics in a large, diverse cohort of patients with ATTRm amyloidosis.
Methods: Adult patients (N = 172) with Stage 1 or Stage 2 ATTRm amyloidosis who had polyneuropathy were screened and enrolled across 24 investigative sites and 10 countries in the NEURO-TTR trial ( www.
Article Synopsis
- Hereditary transthyretin amyloidosis is a severe genetic condition caused by mutations that lead to misfolded transthyretin proteins, resulting in harmful amyloid buildup in various organs, potentially leading to organ failure and death.
- An international phase 3 trial evaluated the effectiveness of inotersen, an antisense oligonucleotide that reduces the production of transthyretin, in improving nerve function and quality of life for patients with ambulatory stages of the disease.
- Results showed significant improvements in both nerve function (mNIS+7 score) and quality of life (Norfolk QOL-DN score) for patients receiving inotersen compared to those on
Background: The objective of the present study was to evaluate the long-term safety and efficacy of tafamidis in treating hereditary transthyretin amyloid polyneuropathy.
Methods: A prospectively planned interim analysis was conducted on an on-going, phase III, open-label extension study following an 18-month, randomized, controlled study and 12-month, open-label extension study in ATTRV30M patients and a single-arm, open-label study in non-ATTRV30M patients. Thirty-seven ATTRV30M patients received placebo for 18 months, then switched to tafamidis and 38 ATTRV30M patients and 18 non-ATTRV30M patients continuously received tafamidis from day 1, up to 6 years.
Focal hypokalemic paralysis: report of 2 cases and review of the literature.
J Clin Neuromuscul Dis
September 2012
Hypokalemia is one of the most frequent electrolytic disturbances encountered in clinical practice. It usually presents with symmetrical generalized muscle weakness and, on occasions, with arrhythmias. There are scarce reports of cases presenting with asymmetric or focal weakness in the literature and no systematic reviews on the subject.
View Article and Find Full Text PDFA case is reported in which an abdominal protrusion appeared in relation with a zosteric eruption at 11th dorsal dermatome. The motor deficit in zoster is unusual (2-3% in the reported series) and generally recognized when the extremities are affected. The frequency with which the abdominal muscles are involved is estimated to be around 0.
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