EURO-NMD supports Rare Disease Day and celebrates the strength of our patients and the dedication of our researchers and clinicians. Patients with Rare Neuromuscular Diseases deserve increased visibility and need treatments to help and, whenever possible, cure their conditions. We have gone a significant way since the initial steps of the 1983 Orphan Drug Act in the USA and the 2009 Recommendation of the EU Council on an action in the field of rare diseases. However, we still only have specific treatments for 5% of our conditions that are genetic in 80% of cases, of which 70% start in paediatric ages. Our patients need continued support and research and are willing to participate and assist the scientific community. The support of the European Commission for this has been pivotal (read about it here). That effort now needs to gain visibility in every Member State’s National Health Service so that no rare neuromuscular disease patient is left behind.
We are stronger together!