Manuel S.
- Country
- France
- Department
- Service Maladies Métaboliques Pédiatriques
Research Overview
- A Phase 1/2 Open-Label Extension Study to Evaluate the Long-Term Safety and Clinical Activity of mRNA-3927 in Participants with Propionic Acidemia
- Efficacy and Safety of Pegtibatinase Added to Standard of Care in Participants with Classical Homocystinuria Due to Cystathionine Beta Synthase Deficiency
- Phase 1/2 Study of Subcutaneous Pegtibatinase (TVT‑058) for Safety, Tolerability, PK/PD and Clinical Outcomes in Patients with Classical Homocystinuria (COMPOSE)
Data & Insights
Has run more than one trial at 11 of 26 partner sites.
Specializations
Metabolic Disorders
The investigator specializes in treating rare metabolic conditions, with particular emphasis on propionic acidemia (PA) and methylmalonic acidemia (MMA). Their research focuses on innovative therapeutic approaches for these inherited metabolic disorders.
- Propionic Acidemia Management
- Methylmalonic Acidemia Treatment
- Metabolic Disease Events (MDEs)
Clinical expertise extends to addressing enzyme deficiencies, particularly methylmalonyl-CoA mutase (MUT) deficiency.
Therapeutic Applications
The investigator explores novel therapeutic approaches using mRNA-based treatments, specifically studying the applications of mRNA-3927 and mRNA-3705 for metabolic disorders.
- Intravenous Therapy Administration
- Long-term Safety Monitoring
- Treatment Efficacy Assessment
Focus includes evaluating therapeutic responses and monitoring treatment outcomes in patients with inherited metabolic conditions.
Clinical Management
The investigator's work encompasses comprehensive patient care strategies for managing rare metabolic disorders, with emphasis on standard of care optimization and safety monitoring.
- Safety and Tolerability Assessment
- Treatment Response Evaluation
- Long-term Patient Monitoring
Expertise includes developing protocols for managing metabolic crisis events and evaluating therapeutic interventions.
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