Marika P.
- Country
- Italy
- Department
- Department of Women's, Child and Public Health Sciences
Research Overview
- Long-term Safety and Efficacy of Weekly Intravenous BMN 351 in Participants with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping: An Open-Label Extension Study
- Efficacy and Safety of DYNE-101 in Participants with Myotonic Dystrophy Type 1: A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study
- Evaluation of Safety, Tolerability, and Efficacy of DYNE-251 in Duchenne Muscular Dystrophy Patients Amenable to Exon 51 Skipping
Data & Insights
Has run more than one trial at 17 of 41 partner sites.
Specializations
Muscular Dystrophy Research
The investigator specializes in groundbreaking research focused on Duchenne muscular dystrophy, with particular emphasis on innovative therapeutic approaches involving exon skipping mechanisms. Their work encompasses studies targeting both exon 44 and exon 51 amenable mutations.
- DMD Treatment Development
- Exon Skipping Therapeutics
- Muscle Tissue Analysis
Myotonic Dystrophy Studies
Extensive research activities in myotonic dystrophy type 1 (DM1) therapeutics, investigating multiple novel treatment approaches. The investigator's work includes evaluation of various therapeutic compounds targeting DM1-specific pathways.
- DM1 Therapeutic Development
- Muscle Function Assessment
- Novel Treatment Modalities
Neuromuscular Disorders
The investigator conducts research in spinal muscular atrophy (SMA), focusing on advanced therapeutic interventions. Their work includes investigation of intrathecal treatments and post-treatment outcomes in pediatric populations.
- SMA Treatment Innovation
- Pediatric Neuromuscular Care
- Treatment Response Assessment
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