Author: Administrator

The ProDGNE consortium is pleased to invite you to the ProDGNE Kick-Off Webinar. In this webinar we will present ProDGNE, a 3 year transnational pre-clinical research project which brings together patients and researchers from Europe and Canada to develop an innovative therapeutic compound to treat GNE Myopathy (GNEM), a rare genetic muscle disease affecting young adults. Speakers: - Fabrizio Pertusati, School of Pharmacy and Pharmaceutical Sciences, Cardiff University, UK - Hanns Lochmüller, University of Ottawa Faculty of Medicine and The Ottawa Hospital Department of Medicine, Canada - Michela Onali, Gli Equilibristi HIBM, Italy - Paula Videira, Faculdade de Ciências e…

SMA NBS Alliance published a whitepaper and call of action on the need for newborn screening for Spinal Muscular Atrophy (SMA). More information you can find on https://www.sma-screening-alliance.org/

EAMDA has 2 new advocacy experts in medicines research, development and therapeutic innovation! Jana Popova, EC Member of EAMDA and member of the Bulgarian Association for Neuromuscular Diseases and Arabela Acalinei, secretary of EAMDA and President of Neuro Move CMT Association in Romania are both graduates of the European Patients’ Academy on Therapeutic Innovation (EUPATI). EUPATI is a 15 months intensive training course that has been specifically designed to meet the needs of patients and patient advocates who have the motivation and skill set to acquire and apply expert-level knowledge across the whole spectrum of the medicine research and development…

There is an updated version of 'Duchenne and you' website available for you to visit at https://duchenneandyou.eu/ Welcome to visit the site and get useful information on DMD (articles & videos included).

European Reference Networks (ERNs) is formed by experts in rare diseases. All ERNs provided their opinion on the priorities and contraindications for patients with a rare disease within their network. The results are summarized in this document and were discussed during the internal ERN-Coordinator group meeting of January 27th, 2021. For more please read the document: 20210205_summary_ERN_COVID-19_Vaccination_DEFDownload

This guidance document is intended for clinicians caring for COVID-19 patients during all phases of their disease (i.e. screening to discharge). This update has been expanded to meet the needs of front-line clinicians and promotes a multi-disciplinary approach to care for patients with COVID-19, including those with mild, moderate, severe, and critical disease. The following sections are entirely new: COVID-19 care pathway, treatment of acute and chronic infections, management of neurological and mental manifestations, noncommunicable diseases, rehabilitation, palliative care, ethical principles, and reporting of death; while previous chapters have also been significantly expanded. WHO-2019-nCoV-clinical-2020.5-engDownload