Pascale D. L.
- Country
- France
- Department
- Pediatric Metabolic Diseases
Research Overview
- Safety and Efficacy Evaluation of Intravenous GLM101 in Patients with PMM2-CDG: A Phase 2 Open-Label Extension Study
- Phase II Multicentric Trial Assessing Efficacy and Safety of Glycerol Phenylbutyrate in Pyruvate Dehydrogenase Deficiency Patients
- Efficacy and Safety Evaluation of Intravenous GLM101 in PMM2-CDG: A Phase 2b, Multicenter, Double-Blind, Randomized, Placebo-Controlled Study
Data & Insights
Has run more than one trial at 8 of 15 partner sites.
Specializations
Metabolic Disorders
The investigator specializes in studying pyruvate dehydrogenase deficiency, a rare metabolic condition affecting cellular energy production. Their research focuses on developing therapeutic approaches for managing this challenging metabolic disorder.
- Enzymatic Deficiency Treatment
- Energy Metabolism Disorders
- Mitochondrial Disease Management
Key interests include evaluating innovative therapeutic strategies for symptom management, particularly addressing fatigue in metabolic disorders.
Therapeutic Innovation
The investigator explores novel therapeutic applications of glycerol phenylbutyrate in metabolic conditions. Their work encompasses evaluating treatment efficacy and safety profiles in rare disease populations.
- Novel Drug Applications
- Treatment Safety Assessment
- Clinical Efficacy Evaluation
Research efforts concentrate on developing targeted treatments for rare metabolic disorders and their associated symptoms.
Clinical Research
The investigator conducts multicentric clinical trials focusing on rare metabolic conditions. Their research methodology emphasizes prospective evaluation of therapeutic interventions.
- Prospective Trial Design
- Multicenter Research
- Patient Outcome Assessment
Expertise includes conducting systematic evaluations of treatment outcomes in specialized patient populations.
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