María del Mar G. R.
- Country
- Spain
- Department
- Neurology
Research Overview
- Phase 3 Open‑Label Study of Intrathecal BIIB115 in Patients Aged 15–60 Years with Spinal Muscular Atrophy, Treatment‑Naïve or Previously Treated with Risdiplam
- Open-Label Extension Study of Dexamethasone Sodium Phosphate in Patients with Ataxia Telangiectasia Following IEDAT-04-2022 Participation
- 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 29 of 65 partner sites.
Specializations
Neuromuscular Disorders
The investigator specializes in treating Spinal Muscular Atrophy (SMA), conducting research into various therapeutic approaches including novel drug delivery systems and combination treatments.
- Type 3 SMA treatment optimization
- Advanced drug delivery mechanisms
- Combination therapy approaches
Their work encompasses both adult and pediatric SMA populations, with particular attention to ambulatory patients.
Rare Genetic Conditions
Focused on therapeutic interventions for Ataxia Telangiectasia (A-T), the investigator evaluates novel treatments targeting neurological symptoms and CNS manifestations.
- Neurological symptom management
- Long-term treatment outcomes
- Pediatric A-T interventions
Their research emphasizes improving neurological function and quality of life in A-T patients.
Muscular Dystrophy
The investigator conducts research in Duchenne Muscular Dystrophy (DMD), focusing on innovative therapeutic approaches and treatment safety assessment.
- Intravenous therapy evaluation
- Long-term safety monitoring
- Pediatric DMD treatment
Their work specifically targets therapeutic interventions for male pediatric patients with DMD.
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