News of the creation of a national registry of people with DMD and BMD in Japan, named Remudy (Muscular Dystrophy Registry (Remudy))and described by Nakamura et al, has been published in the Orphanet Journal of Rare Diseases . This registry was developed in collaboration with TREAT-NMD and includes Japanese male patients with DMD and BMD, most of whom are under 20 years of age. The registry also censuses the type of genetic mutation of patients, their walking status, steroids and cardiac medications taken, ventilation and non-invasive support, and whether patients require corrective surgery for scoliosis. The authors also determined that 9.8% of patients \"were eligible for clinical trials based on 'exon kipping' 51.\" The authors believe that the registry contains sufficient information for recruiting patients for clinical trials, as well as for standardizing care for people with DMD and BMD. In addition, the authors believe that this registry, the first collaborative effort with an international registry, will be able to facilitate \"synchronization of clinical drug developments in Japan with those underway in other countries.<\/p>","protected":false},"excerpt":{"rendered":"
La notizia della creazione di un registro nazionale delle persone affette da DMD e BMD in Giappone, denominato Remudy (Registro della Distrofia Muscolare (Remudy))e descritto da Nakamura et al., \u00e8 stata pubblicata sull\u2019Orphanet Journal of Rare Diseases . Tale registro \u00e8 stato sviluppato in collaborazione con TREAT-NMD e comprende i pazienti giapponesi di sesso maschile […]<\/p>\n","protected":false},"author":4,"featured_media":0,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_et_pb_use_builder":"","_et_pb_old_content":"","_et_gb_content_width":"","footnotes":""},"categories":[1],"tags":[],"class_list":["post-1017","post","type-post","status-publish","format-standard","hentry","category-senza-categoria"],"yoast_head":"\n