Area of research
Neurology · Physiology
Research interest
Research interests include Medicine, Internal medicine, Pediatrics, Disease, Polyneuropathy, and Amyloidosis.
Five-Year Results With Patisiran for Hereditary Transthyretin Amyloidosis With Polyneuropathy
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)
Congenital myasthenic syndromes in adults: clinical features, diagnosis and long-term prognosis
ORAI1 inhibition as an efficient preclinical therapy for tubular aggregate myopathy and Stormorken syndrome
Efficacy and Safety of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease After 97 Weeks
Safety and efficacy of avalglucosidase alfa versus alglucosidase alfa in patients with late-onset Pompe disease (COMET): a phase 3, randomised, multicentre trial
A double-blind, placebo-controlled, randomized trial of PXT3003 for the treatment of Charcot–Marie–Tooth type 1A
Long-term safety and efficacy of patisiran for hereditary transthyretin-mediated amyloidosis with polyneuropathy: 12-month results of an open-label extension study
Early cognitive decline after bilateral subthalamic deep brain stimulation in Parkinson's disease patients with GBA mutations