A5036092944- Amyloidosis: Diagnosis, Treatment, Outcomes
- Alzheimer's disease research and treatments
- Parathyroid Disorders and Treatments
- Coagulation, Bradykinin, Polyphosphates, and Angioedema
- Asthma and respiratory diseases
- Sympathectomy and Hyperhidrosis Treatments
- Lymphoma Diagnosis and Treatment
- Immune Cell Function and Interaction
- Gout, Hyperuricemia, Uric Acid
- Microbial Metabolism and Applications
- Heart Rate Variability and Autonomic Control
- Fungal Biology and Applications
- Peripheral Neuropathies and Disorders
- Sarcoidosis and Beryllium Toxicity Research
- Migraine and Headache Studies
- Bacteriophages and microbial interactions
- Fermentation and Sensory Analysis
- Intracerebral and Subarachnoid Hemorrhage Research
- Pharmaceutical studies and practices
- Drug-Induced Adverse Reactions
- Eosinophilic Disorders and Syndromes
- Protein Tyrosine Phosphatases
- Sarcoma Diagnosis and Treatment
- Bone Tumor Diagnosis and Treatments
- Urticaria and Related Conditions
Hospital de Santo António
2001-2025
Administração Regional de Saúde de Lisboa e Vale do Tejo
2025
Centro Hospitalar do Porto
2013-2022
Hospital de Dona Estefânia
2016-2020
IPO Porto
2019
Centro Hospitalar de Lisboa Central
2016-2018
Importance There is a lack of long-term efficacy and safety data on hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN) RNA interference (RNAi) therapeutics in general. This study presents the longest-term to date patisiran for hATTR-PN. Objective To present adults Design, Setting, Participants global open-label extension (OLE) APOLLO randomized clinical trial phase 2 OLE enrolled patients from 43 hospitals or centers across 19 countries between July 2015 August 2017,...
Hereditary transthyretin (ATTRv; v for variant) amyloidosis, also known as hATTR is a progressive and fatal disease associated with rapid deterioration of physical function patients' quality life (QOL). Vutrisiran, subcutaneously administered RNA interference (RNAi) therapeutic that reduces hepatic production transthyretin, was assessed in patients ATTRv amyloidosis polyneuropathy the pivotal HELIOS-A study.The phase 3 open-label study investigated efficacy safety vutrisiran polyneuropathy,...
Background Anti-calcitonin gene-related peptide (CGRP) monoclonal antibodies (mAbs) are considered a safe and well-tolerated option for migraine prophylaxis. However, caution is advised in patients with vascular comorbidities. In hereditary transthyretin amyloidosis (ATTRv amyloidosis), amyloid deposition leptomeningeal vessels has been reported, potentially leading to cerebral angiopathy. The efficacy safety of anti-CGRP this condition have not described. Methods We present three ATTRv...
Patients with hereditary amyloidosis related to transthyretin (ATTRv amyloidosis) treated liver transplant (LTx) often have central nervous system (CNS) manifestations, including cognitive dysfunction. The aim of this study was explore the long-term outcome associated neuropsychological test performance. A retrospective longitudinal review conducted in a cohort 289 ATTRv patients Val30Met mutation (ATTRV30M who underwent assessment (T1) 1-23 years (median = 11) post-LTx and 20-189 months 81)...
Central nervous system dysfunction is common in longstanding hereditary transthyretin amyloidosis (ATTRv) caused by the V30M (p.V50M) mutation. Neuropathology studies show leptomeningeal amyloid deposition and cerebral angiopathy (CAA). Brain MRI widely used assessment of Aβ associated CAA but there are no systematic with brain ATTRv amyloidosis.
Cognitive dysfunction is part of the broad spectrum clinical manifestations in older untreated hereditary transthyretin amyloidosis patients with peripheral polyneuropathy.The objective this study to systematically explore cognitive ATTRV30M whose disease course was modified by liver transplant (LT).A series 269 carriers TTRVal30Met mutation treated LT underwent a neuropsychological assessment. Clinical charts were reviewed identify focal neurological episodes (FNEs), complaints and...
Data on risk factors for uncontrolled asthma in preschool children are controversial. Objective: This study aims to explore the association between clinical and functional parameters lack of control children.Children aged 3-5 years with healthy controls were recruited. A questionnaire was used identify potential asthma, as defined by Global INitiative Asthma criteria. Lung function bronchial reversibility evaluated through impulse oscillometry spirometry. Adjusted odds ratios estimated based...
Transthyretin (TTR) related Familial Amyloid Polyneuropathy presents as a severe sensory, motor and autonomic neuropathy. Tafamidis, an oral drug that stabilizes TTR preventing amyloid deposition, was recently introduced in Europe to delay neuropathy progression ambulatory patients.
OBJECTIVE: Domino liver transplantation (DLT) and de novo familial amyloid polyneuropathy (FAP).
Introduction. The Test for Respiratory and Asthma Control in Kids (TRACK) is a tool to assess asthma control preschool children. This study aims validate the Portuguese from Portugal version of TRACK questionnaire. Methods. A prospective cohort was carried out their psychometric characteristics. Caregivers 141 children under age 5 with symptoms were enrolled. Results. Internal reliability close 0.70 (Cronbach's α). test-retest 0.87. scores different between well, partially, non-controlled...
Abstract Background and Aims Transthyretin (TTR) amyloidosis is a disease characterized by destabilization of the native TTR tetramer. It can be caused aging or due to pathogenic mutations, its diagnosis often missed. Hereditary transthyretin (ATTRv), Val50Met mutation, frequently linked with polyneuropathy but it cause nephropathy too, causing wide range levels albuminuria renal dysfunction. Tafamidis, tetramer stabilizer, has been associated significant decrease in ATTRv. However, not an...