Nuria M. G.
- Country
- Spain
- Department
- Neuromuscular Unit
Research Overview
- Open-label extension study of KL1333 for long-term safety, tolerability, and efficacy in adults with primary mitochondrial disease
- Phase 3 Randomized Double‑Blind Placebo‑Controlled Study of Single Intravenous SGT‑003 Gene Therapy in Ambulant Male Patients with Duchenne Muscular Dystrophy
- A Phase 2 Randomized, Double-blind, Placebo-controlled Study to Evaluate the Effect of EDG-5506 on Safety, Biomarkers, Pharmacokinetics, and Functional Measures in Adults and Adolescents with Becker Muscular Dystrophy
Data & Insights
Has run more than one trial at 92 of 144 partner sites.
Specializations
Muscular Dystrophy Research
This investigator specializes in various forms of muscular dystrophy, with particular focus on Duchenne Muscular Dystrophy (DMD) and Becker Muscular Dystrophy (BMD) therapeutic interventions.
- Gene transfer therapies
- Novel pharmaceutical compounds
- Long-term treatment outcomes
Advanced expertise in evaluating treatments for both ambulatory and non-ambulatory patients with muscular dystrophies.
Inflammatory Muscle Disorders
Focused on investigating treatments for Idiopathic Inflammatory Myopathy (IIM) and related autoimmune muscle conditions.
- Subcutaneous therapeutic applications
- Immunomodulatory treatments
- Long-term safety assessments
Specialized in evaluating novel biological therapies for inflammatory muscle conditions.
Rare Neuromuscular Conditions
Conducts research in rare muscle disorders including Facioscapulohumeral Muscular Dystrophy (FSHD) and Myotonic Dystrophy.
- Congenital myasthenic syndromes
- Progressive muscle weakness treatments
- Therapeutic tolerance studies
Expert in evaluating novel compounds for rare genetic muscle disorders.
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