Francina M. C.
- Country
- Spain
- Department
- Servicio de Neurologia Pediatrica
Research Overview
- Phase 3 Multicenter Randomized Double-Blind Placebo-Controlled Trial of Fordadistrogene Movaparvovec in Duchenne Muscular Dystrophy Patients
- Evaluation of Safety, Tolerability, and Efficacy of DYNE-251 in Duchenne Muscular Dystrophy Patients Amenable to Exon 51 Skipping
- Phase 2 Open-Label Study on Safety and Pharmacokinetics of Doxecitine and Doxribtimine in Thymidine Kinase 2 Deficiency Patients
Data & Insights
Has run more than one trial at 13 of 38 partner sites.
Specializations
Rare Genetic Disorders
The investigator specializes in treating thymidine kinase 2 (TK2) deficiency, focusing on innovative therapeutic approaches using nucleoside combinations.
- Pyrimidine nucleoside therapy
- Metabolic pathway interventions
Their work explores treatment options for rare genetic conditions affecting cellular metabolism.
Neuromuscular Disorders
Primary focus on Duchenne Muscular Dystrophy (DMD), investigating multiple therapeutic approaches including gene therapy and novel drug compounds.
- Gene therapy applications
- Exon skipping therapeutics
- Long-term treatment strategies
Expertise in evaluating progressive muscle disorders and implementing innovative treatment protocols.
Motor Neuron Diseases
Active involvement in treating Spinal Muscular Atrophy (SMA) through advanced therapeutic interventions.
- Intrathecal therapy administration
- Long-term safety monitoring
- Neurological disease management
Specialized in evaluating therapeutic outcomes in progressive neurological conditions.
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