Author: Michal Rataj

EAMDA Scientific Conference 2025, held online on 27–28 November 2025, brings together key stakeholders in the neuromuscular field, including clinicians, researchers, healthcare providers, patient organizations, individuals with lived experience, and representatives from the pharmaceutical sector, to discuss the latest advancements in the neuromuscular field. Over two days, the online conference will explore therapeutic developments, innovations in newborn screening, multidisciplinary care models, rehabilitation approaches, and collaborative initiatives driving change across Europe. *This online event will be recorded. Your participation constitutes consent to the use of your image and voice for the purposes of documenting and reporting on the conference. If you…

EAMDA extends its appreciation to Roche, Biogen and Alexion Pharmaceuticals, Inc. for their continuous commitment and generous support in organizing “Advancing Together: Shaping a Brighter Future for NMD Patients” Workshop in Ljubljana. With their partnership, we successfully brought together patients, patient representatives, experts, and other interested stakeholders to present and discuss on diagnostics, patient care, research, and patient empowerment in the NMD community. Thank you for your commitment to help us make a lasting impact!

On 25–26 October 2024, EAMDA hosted the “Advancing Together: Shaping a Brighter Future for NMD Patients” Workshop in Ljubljana, Slovenia—a capacity-building event, dedicated to our member organizations. We brought together patients, patient representatives, pharma partners, and other interested stakeholders for two days of insightful presentations and discussions covering essential topics, including diagnostic and newborn screening, care of NMD patients, research and clinical trials. Participants also had a unique opportunity to deepen their understanding of Euro-NMD ERN and the extensive range of activities it offers. There is still a significant need for increased involvement from patient representatives, especially from Eastern Europe,…

US Food and Drug Administration (FDA) has approved delandistrogene moxeparvovecrokl (also known as SRP 9001) for the treatment of ambulatory children (those who are able to walk unassisted) aged 4 through 5 years with Duchenne muscular dystrophy (DMD) and a confirmed mutation in the DMD gene (23 June 2023). See more details in the document below. Roche Community Letter - SRP-9001 FDA ApprovalDownload  

The next intake of the EUPATI Patient Expert Training is now open for registrations! The EUPATI Patient Expert Training Programme is a training programme about the medicines’ development process. It covers the entire lifecycle of medicines research and development (R&D), from the design and execution of clinical trials to regulatory processes and Health Technology Assessment (HTA). In addition to detailed information on each step of the process, the training also describes how patients can be involved at each stage. The overall objective of the programme is to obtain a thorough understanding of the medicines R&D process, the patients’ role within…

We are pleased to announce a spinal muscular atrophy webinar as part of our activities in SMA Awareness Month. Tuesday, August 30th 2022 17:00 to 19:00 Central European Time Online Please save the date Zoom link registration below: Register