Lorenzo M.
- Country
- Italy
- Department
- UOC Neurologia 4
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
- Phase 3 Randomized Quadruple-Blind Study on Batoclimab Efficacy and Safety in Adult Generalized Myasthenia Gravis Patients
- Efficacy, Safety, and Tolerability of NMD670 in Ambulatory Adults with Type 3 Spinal Muscular Atrophy: A Phase 2 Randomized, Double-Blind, Placebo-Controlled Study
Data & Insights
Has run more than one trial at 33 of 100 partner sites.
Specializations
Neuromuscular Disorders
The investigator specializes in treating various forms of myasthenia gravis, including both generalized and congenital variants. Their research extends to innovative therapeutic approaches for DOK7 Congenital Myasthenic Syndrome.
- Generalized Myasthenia Gravis (gMG)
- Congenital Myasthenic Syndromes
Their work encompasses novel therapeutic interventions for neuromuscular junction disorders.
Muscular Dystrophies
The investigator focuses on treating progressive muscle disorders, particularly Facioscapulohumeral Muscular Dystrophy (FSHD) and LMNA-related muscular disorders.
- Facioscapulohumeral Muscular Dystrophy
- Laminopathies
Their expertise includes evaluating innovative treatments for progressive muscle weakness conditions.
Motor Neuron Diseases
The investigator conducts research in spinal muscular atrophy, with particular emphasis on Type 3 SMA in ambulatory adult patients.
- Type 3 Spinal Muscular Atrophy
- Adult-onset Motor Neuron Disorders
Their work focuses on therapeutic interventions to maintain muscle strength and function.
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