Raul J. M.
- Country
- Spain
- Department
- Neurology
Research Overview
- Efficacy and Safety of Surlorian in Adults with Autosomal Dominant RYR1-Related Myopathy: A Phase 2, Randomized, Double-Blind, Placebo-Controlled Trial
- Efficacy and Safety of DYNE-101 in Participants with Myotonic Dystrophy Type 1: A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study
- Open-label extension study of KL1333 for long-term safety, tolerability, and efficacy in adults with primary mitochondrial disease
Data & Insights
Has run more than one trial at 172 of 292 partner sites.
Specializations
Neuromuscular Disorders
This investigator specializes in research concerning myasthenia gravis, with particular focus on both generalized and ocular forms. Their work encompasses studies of novel treatments including efgartigimod, nipocalimab, and zilucoplan.
- Generalized Myasthenia Gravis
- Ocular Myasthenia Gravis
- Seronegative Myasthenia Gravis
Muscular Dystrophies
The investigator conducts research in various forms of muscular dystrophy, including Becker Muscular Dystrophy and Facioscapulohumeral Muscular Dystrophy. They evaluate innovative therapeutic approaches for these conditions.
- FSHD Type 1
- Becker Muscular Dystrophy
- Motor Function Assessment
Neuropathies
Their expertise extends to inflammatory and hereditary neuropathies, with significant work in Chronic Inflammatory Demyelinating Polyneuropathy and Multifocal Motor Neuropathy. The research includes studies of Charcot-Marie-Tooth Disease.
- CIDP Treatment Approaches
- Motor Neuropathy Management
- Peripheral Nerve Disorders
Motor Neuron Diseases
The investigator focuses on therapeutic interventions for Amyotrophic Lateral Sclerosis and Spinal Muscular Atrophy, investigating novel treatment approaches and disease-modifying therapies.
- ALS Treatment Studies
- Type 3 SMA Research
- Motor Function Preservation
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