Dr. Eduardo Tizzano, director of the Clinical and Molecular Genetics Area and head of the group of Genetics Medicine of Vall d'Hebron Institute of Research (VHIR) and Dr. Francina Munell, from de Pediatric Neurology Service and head of the research line of Pediatric Neuromuscular Disorders of VHIR, together with other professionals of pediatric neurology, rehabilitation, neurophysiology, clinical genetics and VHIR researchers obtained the recognition of http://bit.ly/2ifv8ct" INC Research, the company that coordinates the clinical trial of a new drug to treat the pediatric spinal muscular atrophy, Nusinersen. Nusinersen is a proprietary antisense oligonucleotide that modulates alternate splicing of the SMN2 gene, involved in the disease. Vall d'Hebron was selected from more than 1.000 projects ongoing of this company as one of the 130 sites committed to the development of new drugs to treat the patients.The aim of the project in which the site participates is proving the effectiveness of Nusinersen, developed by Ionis Pharmaceuticals to treat pediatric spinal muscular atrophy, a neuromuscular pathology, with a genetic condition, which affects motor neurons. This disease classifies in three principal groups, the acute form type I or Werdnig-Hoffman disease, which affects 1 in 6.000 newborns and results in death of almost all the cases at two or three years old, the intermediate form type II and the mild form of the disease, the type II form or Kugelberg Welander disease.Important participation of Vall d'HebronVall d'Hebron was one of the 30 centers participating in this clinical trial, which has a high complexity because of the characteristics of the children involved. The study ENDEAR was designed as a randomized, double-blind study in patients with type I form of the disease. A third part of the patients received placebo. INC Research recognized the work of the professionals in the study and their coordinated work and adherence to the protocol. Dr. Tizzano assures that it is "a recognition to the whole team, who worked hardly". "It has been a pleasure working with them, they are excellent and the study would not have been possible without them", he assured, highlighting that things like this "helps us to make further progress".The first results of the trial are encouraging and the start of a new phase study has been already announced, opened to all the patients who participated in the previous phase and others not participating. Vall d'Hebron has already started to treat the first patient in the new clinical trial.