JWMDRC

🧬 Undiagnosed Day 2026 – Where diagnosis becomes hope On 30 April in Gdańsk, ERDERA and partners host a key event on collaboration & technology in rare diseases, following a clinician round table on 29 April. 📅 29–30 Apr 2026 🔗 erdera.org/event/undiagno… #UndiagnosedDay

Children need cancer treatments designed for their bodies and needs. Our Paediatric Therapeutic Development Workshops pinpoint the most promising therapeutic targets for future drugs and trials, in cancers with the greatest unmet needs. Learn more: t.ly/umN1O

Active8 are looking for young people with physical disabilities to join their fully funded programme this Spring/Summer 2026. The programmes, based in Cornwall, include everything from creative workshops to adventure activities! Find out more here 👉 active8online.org

📣 Register now for Undiagnosed Day 2026 (29–30 April) A clinician-led meeting on phenotype-led diagnosis, featuring live case discussions and multidisciplinary exchange. 📍Gdansk, Poland + online 🔗erdera.org/event/undiagno…

Congratulations to the team for a new paper published 👏 🧫By combining advanced co-culture systems, secretome profiling, and 3D engineered muscle models, we investigated how #Duchenne muscular dystrophy (DMD)-derived FAPs inhibit myogenesis. bit.ly/3PE1Hpv

Check out the latest PaLaDIn Newsletter March 2026 to catch up on recent work on the project and developments in the Interactium platform! 👀 linkedin.com/pulse/paladin-… @eu_paladin

📣 Group Discussions at the European Conference on Rare Diseases & Orphan Products 2026 📣 As an associate partner of #ECRD2026, we invite in-person attendees to submit proposals for small-group discussion topics. 🗓 Deadline: 17 April 2026, 18:00 CET 🔗 lnkd.in/dD6A_Pmm

🌍 European Reference Networks (#ERNs) connect rare disease experts across Europe. Within #ERDERA, ERNs help bring clinicians, researchers & patients together to share knowledge, advance research and improve care where it’s needed most.💙 Find out more: youtu.be/9A0RNKFy7ls

One of our Strand Leads, Prof Michela Guglieri, presenting "gene therapy in muscle diseases" as part of a series of seminars in Padova on neurology topics; “martedì della clinica neurologica” meaning "Tuesdays of the neurology clinic" 😀

Save the Date and plan for the big day with our toolkit! lgmd-info.org/toolkit/

Voting is LIVE! Don't miss your chance to vote for your favorite t-shirt design for World FSHD Day, the Walk & Roll, and general merch. Voting closes Saturday, March 21 at 11:59pm ET surveymonkey.com/r/XYVFTQM

📢Another paper to celebrate! This one, authored entirely by members of the JWMDRC, aims to provide insight into how mobility, age, and diagnosis may affect health-related quality of life in adults with neuromuscular disease👏 neurology.org/doi/pdf/10.121…

We are pleased to announce a new publication "Cracking the Code: Genotype–Phenotype Correlation Models in Sarcoglycanopathies" which aimed to establish accurate genotype–phenotype correlations for LGMDR3, LGMDR4, and LGMDR5 👏 bit.ly/4lGc1sL #LGMD

For more info, visit: lgmd-info.org

Help us, help you, recruit for your study! To manage study requests, and make sure we share them with the correct audiences, we've create a new Study Submission Form to aggregate and disseminate. fshdsociety.org/submit-your-st… #FSHDResearch #FSHD

We're attending the #PaLaDIn project General Assembly in Munich at the impressive Carl Friedrich von Siemens Stiftung Hunter's Lodge. The consortium will be reviewing the project progress and planning for the following year. @eu_paladin @TREAT_NMD @PromptlyHealth

To mark #RareDiseaseDay 2026, the short film of our global #RaisingYouthVoices2026 event was released! 🎥 Learn more about the issues that are driving young members of our community to fight for a more equitable future.⬇️

We’re excited for Muscular Dystrophy UK’s Birmingham Information Day on Sat 14 March - a free event for adults & parents of children with muscle‑wasting conditions. We’re representing JWMDRC & sharing the work of our Registries Team. Sign up: bit.ly/4rj0CQM @MDUK_News

Five years after the Rare2030 recommendations and one year after the WHA called for a Global Action Plan, rare disease policy is both a moral imperative and a strategic investment. Be part of this momentum. 👉 go.eurordis.org/register-x

*Seeking 80 Participants: the MOVE Pediatric Study* The study seeks to increase understanding of Early-onset FSHD & refine clinical trial design for early-onset/severely affected individuals. Stipend provided. This study is free & requires onsite visits. clinicaltrials.gov/study/NCT06847…