Márcio C.
- Country
- Portugal
- Department
- Neurophysiology Department
Research Overview
- Phase 3 Randomized Double-Blind Placebo-Controlled Study of Acoramidis Hydrochloride for Transthyretin Amyloidosis Prevention in Asymptomatic TTR Variant Carriers
- A Phase 3, Open-Label, Multicenter, Extension Study of Acoramidis in Patients with Newly Diagnosed Variant Transthyretin Amyloid Cardiomyopathy (ACT-EARLY OLE)
Data & Insights
Has run more than one trial at 29 of 36 partner sites.
Specializations
Amyloidosis Research
This investigator specializes in transthyretin amyloidosis, focusing on preventive therapeutic approaches for individuals with genetic predisposition to this rare condition.
- Early Disease Prevention
- Genetic Variant Analysis
- Pre-symptomatic Treatment
The investigator's work encompasses both cardiac and peripheral nervous system manifestations of amyloidosis.
Genetic Disease Management
The investigator conducts research in hereditary disease prevention, particularly focusing on asymptomatic carriers of pathogenic variants.
- Genetic Risk Assessment
- Preventive Medicine
- Early Intervention Strategies
Their work involves identifying and managing patients with genetic predispositions before disease onset.
Therapeutic Innovation
The investigator explores novel therapeutic approaches in rare genetic disorders, with a focus on disease-modifying treatments.
- Novel Therapeutic Development
- Disease Progression Prevention
- Long-term Disease Management
Their research aims to advance treatment options for genetic conditions affecting multiple organ systems.
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