The EU Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for the extension of the Evrysdi® (risdiplam) European Union marketing authorisation, which would include infants with genetically confirmed diagnosis of SMA Type 1, Type 2 or Type 3 or with one to four SMN2 copies, including from birth to below […]

  We need your help to develop and plan research! MDI strives to support and fund quality research into neuromuscular conditions. However, when planning research, the expertise of people with lived experience of the condition is sometimes missing. It is important that this lived experience is combined with the expertise of researchers. This partnership can […]

About myasthenia gravis They are two main types of myasthenia gravis (MG), generalised and ocular. Generalised MG is the most common type, accounting for 85 per cent of people with MG. It is a rare condition with a global prevalence of 100–350 cases per every one million people. Muscle weakness is the hallmark of myasthenia […]

On 22 June, the US Food and Drug Administration (FDA) granted accelerated approval to Sarepta Therapeutics’ gene therapy SRP-9001 (also known as delandistrogene moxeparvovec-rokl) for Duchenne muscular dystrophy (DMD). Approval was granted for the treatment of children aged between four and five years, with Duchenne muscular dystrophy who can walk unassisted. The FDA’s accelerated approval […]

Are you over 18 years of age, living with a neuromuscular condition and interested in sharing your experiences and challenges of transitioning to adulthood such as leaving secondary school, getting a job, going to post-secondary education, meeting young people socially etc? If yes, we want to hear from you! Moving into adulthood can be a […]

  Researchers around the world are working very hard to find cures and treatments for neuromuscular conditions. Drug development takes years and at times studies that show promising results throughout the different clinical trial phases may fail at the last hurdle. Some promising studies on the horizon are described below and we will be watching […]

MDI strives to support and fund quality research into neuromuscular conditions. However, in research, we are sometimes missing the expertise of people with lived experience of the condition. MDI are committed to bringing your voice into research, which will help researchers focus on specific elements that will impact the community while explaining their research in […]

One of MDI’s strategic priorities is to support researchers and clinicians to carry out quality research into neuromuscular conditions. An initiative in achieving this is the development of a registry of people in Ireland with a neuromuscular condition. A registry is a database containing information about individuals who are affected by a specific condition. In […]

Call for research participants – creating resources for young people to prepare for transitioning to adulthood Are you 21 to 30 years with muscular dystrophy and interested in sharing your experiences with researchers to assist in creating resources for young people to prepare for transitioning to adulthood? Moving into adulthood can be a hard time […]