Andrés N. O.
- Country
- Spain
- Department
- Servicio de Neurologia
Research Overview
- Phase 3 Randomized Double‑Blind Placebo‑Controlled Study of Single Intravenous SGT‑003 Gene Therapy in Ambulant Male Patients with Duchenne Muscular Dystrophy
- Efficacy and Safety Evaluation of Avalglucosidase Alfa in Treatment-Naïve Pediatric Patients with Infantile-Onset Pompe Disease (IOPD)
- Evaluation of Clinical Outcomes of Nusinersen in Patients with Spinal Muscular Atrophy Post-Onasemnogene Abeparvovec Administration
Data & Insights
Has run more than one trial at 30 of 65 partner sites.
Specializations
Neuromuscular Disorders
The investigator specializes in treating patients with Spinal Muscular Atrophy (SMA) and Duchenne Muscular Dystrophy (DMD), focusing on innovative therapeutic approaches for these genetic conditions.
- Progressive Muscular Disorders
- Genetic Neuromuscular Diseases
- Pediatric Neurology
Particular attention is given to evaluating therapeutic responses in patients with complex neuromuscular conditions.
Treatment Innovation
The investigator works with advanced therapeutic agents including nusinersen and onasemnogene abeparvovec for SMA treatment, as well as givinostat for DMD management.
- Novel Therapeutic Approaches
- Long-term Treatment Outcomes
- Safety and Efficacy Assessment
Focus extends to monitoring long-term therapeutic outcomes in patients with progressive muscular conditions.
Clinical Management
Expertise encompasses the clinical assessment and therapeutic monitoring of patients with complex neuromuscular disorders, emphasizing personalized treatment approaches.
- Treatment Response Evaluation
- Long-term Patient Monitoring
- Safety Protocol Development
Special interest in evaluating treatment efficacy and safety in previously treated patients.
Related Investigators
Researchers in similar fields or departments
Want to know more about this investigator?
We can share extended profile information and contact details.
Is something out of date?
Tell us what needs correcting and we'll update this profile.