Area of research
Neurology · Oncology
Research interest
Research interests include Medicine, Myasthenia gravis, Placebo, Tolerability, Internal medicine, and Eculizumab.
Safety and efficacy of losmapimod in facioscapulohumeral muscular dystrophy (ReDUX4): a randomised, double-blind, placebo-controlled phase 2b trial
104-week efficacy and safety of cipaglucosidase alfa plus miglustat in adults with late-onset Pompe disease: a phase III open-label extension study (ATB200-07)
Safety and efficacy of arimoclomol for inclusion body myositis: a multicentre, randomised, double-blind, placebo-controlled trial
Patients with transthyretin amyloidosis enrolled in THAOS between 2018 and 2021 continue to experience substantial diagnostic delay
Identification of genetic risk loci and prioritization of genes and pathways for myasthenia gravis: a genome-wide association study
Development of a standard of care for patients with valosin-containing protein associated multisystem proteinopathy
Clinical Effects of the Self-administered Subcutaneous Complement Inhibitor Zilucoplan in Patients With Moderate to Severe Generalized Myasthenia Gravis
Epidemiological evidence for a hereditary contribution to myasthenia gravis: a retrospective cohort study of patients from North America
A Genome-Wide Association Study of Myasthenia Gravis
A randomized, double‐blind, placebo‐controlled phase II study of eculizumab in patients with refractory generalized myasthenia gravis
SMA valiant trial: A prospective, double‐blind, placebo‐controlled trial of valproic acid in ambulatory adults with spinal muscular atrophy
Randomized, Double-Blind, Placebo-Controlled, Crossover, Multicenter, Phase II Study of Eculizumab in Patients with Refractory Generalized Myasthenia Gravis (gMG) (S35.004)