Eighteen researchers and clinicians and two representatives of patient organizations (Associazione Italiana Calpaina 3, Italy and Coalition to Cure Calpain3, USA), from 9 countries (France, Germany, Italy, Denmark, Spain, UK, Japan, Brazil and USA), met in Naarden, The Netherlands, from 15–17 September, 2017 to discuss clinical trial readiness for limb-girdle muscular dystrophy type 2A (LGMD2A; OMIM 253600), also classically referred to as calpainopathy. LGMD2A is due to mutations in the CAPN3 gene encoding a calcium-dependent cysteine protease named calpain 3 []. This disease is characterized by slowly progressive muscle weakness affecting selectively the musculature of both girdles. There is no effective treatment for this disease to date. The emergence of novel therapeutic approaches in the field, such as gene therapy, has prompted a much awaited discussion among physicians and researchers about the readiness for clinical trials in calpainopathy.