Therapies for Neuromuscular Diseases

touchNEUROLOGY met with Dr Teresa Coelho (Unidade Clinica de Paramiloidose, Hospital de Santo Antonio, Porto, Portugal) to discuss new therapies for the treatment of hereditary ATTR amyloidosis. Hereditary ATTR amyloidosis is a progressive and fatal disorder caused by variant transthyretin deposition as amyloid. The interview discusses an overview of tafamidis, patisiran, and inotersen, and their […]

touchNEUROLOGY met up with Prof. Giuseppe Lauria Pinter (University of Milan, Milan, Italy) to discuss the efficacy and outcome measures of a phase 2 study investigating guanabenz for the treatment of amyotrophic lateral sclerosis. The abstract entitled: ‘The unfolded protein response in Amyotrophic lateral sclerosis: Results of a Phase 2 Trial’ was presented at the […]

touchNEUROLOGY met with Renato Mantegazza (Department of Neuroimmunology and Neuromuscular Diseases, Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta, Milan, Italy) to discuss the challenges that arise in treating patients with myasthenia gravis. The abstract entitled: ‘Safety and Tolerability of Efgartigimod in Patients with Generalized Myasthenia Gravis: Phase 3 ADAPT Study Results’ was presented at the 7th […]