Proud to partner with @eurordis for #ECRD2026, Europe’s largest patient-led rare disease event. Join us in Prague & online, 3–4 June 2026. Poster abstracts now open! 👉 www.rare-diseases.eu/posters

Applications are open to join the PaLaDIn Patient and Caregiver User Group (PCUG). We're looking for #patients and #caregivers with experience of a #neuromuscular disease to join and contribute. Further information is available here: bit.ly/47V9DrS

We have a strong attendance at #WMS2025 on its 30th birthday, and this is the last year for our Centre Director, Prof. Volker Straub, as President. Volker gave the Congress Welcome this morning, inviting everyone to have an enjoyable and productive few days here in Vienna

📢New projects feature in the #LGMD News Autumn edition. Produced for the community by The Speak Foundation, this is a great resource for members of the LGMD community🙌 See page 14 for an article about new projects at our centre! 📰Sign up here: bit.ly/4pWGsNl

Today we're supporting #LGMD Awareness Day! 🙌 While we can't reverse LGMD yet, proper care, support, and therapies can make a world of difference. Let's advocate for accessible care for all living with LGMD. #LGMDday2025 #lgmdawarenessday

Today at the FSHD Annual Engagement Day experts from NuTH are giving useful talks on pain management, sleep issues and exercise prescription👩‍⚕️ We're also getting research updates from the lab team on FSHD mechanisms, lab studies and imaging projects👨‍🔬 #FSHD

Today we're supporting international myotonic dystrophy awareness day! #MyotonicDystrophy (DM) is an inherited multisystem condition that mainly causes progressive muscle loss, weakness and myotonia. Join us in raising awareness and supporting research: bit.ly/41Wng82

⭐£22M NIHR investment to strengthen UK health & life sciences innovation We are delighted that @nihr-io.bsky.social at @newcastleuni.bsky.social has been awarded £22M by @nihr.bsky.social to continue research identifying emerging medicines, diagnostics, devices & digital tech 👉 ow.ly/ooyc50WPtFj

🎈 On World Duchenne Awareness Day, we stand with the #Duchenne & #Becker Muscular Dystrophy community. At JWMDRC, we’re committed to advancing care, research & inclusion—because Duchenne is a lifelong journey. #WDAD2025

FSHD Annual Engagement Day – 25 Sept, Newcastle 🔬 Research updates 🩺 Clinical sessions 👩‍⚕️ 1:1 consultations 💬 Last year: “high quality, interactive & informative” Book now: rb.gy/gabmw7 #FSHD

Our director Volker Straub receiving the LGMD Innovator award from Kelly Brazzo from CureLGMD2i at the International LGMD Conference in Orlando on 19th July 👏🥳 #LGMD

We're looking for children & adults with Nemaline Myopathy to join a study to collect data on the natural progression of the condition. Help us understand Nemaline Myopathy to be ready for therapeutic developments See more at bit.ly/3HeoRPc #NemalineMyopathy #RareDisease

🔍 Want to model rare disease progression using #ArtificialIntelligence & #ML? Join a PhD at Newcastle Uni tackling FSHD with #bioinformatics + #MachineLearning. Part of LifeArc ARDT’s £12m UK-wide initiative. To apply: bit.ly/4eVKUqe

Join us for the FSHD Engagement Day in Newcastle on 25 Sept 2025! Connect with others, hear from experts & explore key aspects of life with FSHD. Book your place: rb.gy/gabmw7 #FSHD #FSHDUK #FSHDEurope #WorldFSHDDay

Now for our big news... I'm delighted to announce that the UK FSHD Patient Registry has now 🌟 RELAUNCHED 🌟 onto our new bespoke registry platform, with improved questionnaires and features! Visit our website to learn more and sign up today! 👀 www.fshd-registry.org.uk 👀 #FSHD #WorldFSHDday

We were fortunate to attend the @fshdsociety.bsky.social International Research Congress and FSHD Europe Patient Connect meetings last week. These fantastic events brought together the FSHD community across Europe to make new connections and share research advancements #WorldFSHDday

🍊 Today is World FSHD Day! 📅 Held on June 20th to raise public awareness of #FSHD 🧬 Our Patient Registry helps advance research and development of treatments, therapies, and care for all those diagnosed with FSHD in the UK. 💻 Visit our newly revamped website to learn more: fshd-registry.org.uk

The JWMDRC is proud to support the launch of the PaLaDIn Interactium data platform tender! Bids are open till 17 July—help us shape the future of rare disease data infrastructure. More info 👉 project-paladin.eu/paladin-inte...

Chris Higgins, CEO of Rare Disorders New Zealand, visited us to explore advances in rare disease care & research. We discussed #neuromuscular projects like Care-DMD, PaLaDIn & MYO-Seq. His visit sparked ideas for strategic collaboration across regions. 🇳🇿🇬🇧 #RareDisease #DMD #MYO

We are pleased to announce a new paper published on a study aimed to characterise a large cohort of paediatric and adult patients with Becker muscular dystrophy (#BMD) to inform clinical care and the development of standards of care guidelines 👏 link.springer.com/content/pdf/...

We attended the MDUK Patient Day in Northern Ireland to talk about our vital work in neuromuscular diseases. Helen Walker gave a talk on the importance of patient registries, and Abby Scott and Adam Kerr spoke to delegates about the #BMD Hub and #DMD Care UK.

Dr Adam McDiarmid, Consultant Cardiac Transplant Physician & Chair of DMD Care UK's cardiac MRI task force, speaks today at #BCS2025 on cardiac imaging in Duchenne. Great to see this vital topic highlighted at such a high-profile event! 👏 #DMD #CardiacCare

Congratulations to conect4children who have just had a paper published in the Journal of Medical Internet Research 'The potential to leverage real-world data for paediatric clinical trials: A proof of concept study' 👏 www.jmir.org/2025/1/e72573

As part of the celebrations to mark 25 years of the Centre for Life our Centre Director, Professor Volker Straub, gave a lecture focusing on recent successes and developments in the #neuromuscular field at the ‪JWMDRC: youtube.com/post/UgkxdcG...

📢 Join us this September in Newcastle for a free #BMD Patient Day! Organized by BMD Hub & TREAT_NMD, the event includes expert talks, workshops & support. Connect with others who understand your journey. Register now 👉 wkf.ms/4jvcKLL

Happy #ClinicalTrialsDay from JWMDRC! 🎉 Today, we celebrate the researchers, healthcare professionals, and amazing volunteers who drive medical progress. Your dedication is shaping the future of treatment and care. Thank you for making a difference! 💙 #ICTD2025

INSIGHTS Public Lecture: The Newcastle way: Leading advancements in #neuromuscular research By Professor Volker Straub. Date: 15 May Time: 17:30 - 18:30 Location: Curtis Auditorium, Herschel Building, Newcastle University @newcastleuni.bsky.social www.ncl.ac.uk/university-e...

Congratulations to Rasya Gokul Nath for winning one of the three poster prizes at the UK Neuromuscular Translational Research Conference for her excellent work on "A spatial transcriptomic approach to understanding Duchenne muscular dystrophy" 🙌🫡

The JWMDRC enjoyed the recent UK Neuromuscular Translational Research Conference at Newcastle University, and had a strong presence to present and discuss our work

We are proud to announce that Physiotherapy & occupational therapy clinical guidelines have been published in the European Journal of Paediatric Neurology 🙌 developed by Anna Mayhew from the JWMDRC and DMD Care UK's physiotherapy and occupational therapy working group. bit.ly/4jkkEaK

The BMD Hub has launched a UK focused survey for those living with Becker Muscular dystrophy to help us understand current care practices and identify gaps and areas for improvement. Learn more and take part in the survey here: tinyurl.com/ywbdtd6j #BMD

The Highly Specialised Service for rare neuromuscular disorders in Newcastle is organising an info day for people living with #LGMD, in person or remotely. This will be a mixture of talks from experts in research, clinical care, and interactive workshops. bit.ly/3RBg8YU

Very proud to share our last publication to help improving understanding of the assessment of disease progression in SMA @jwmdrc.bsky.social @mdukcharity.bsky.social @curesma.bsky.social www.nmd-journal.com/action/showP...

DMD Care UK are running online, live care workshops for health care professionals on 28th March! 👀 Find out more & register here: jwmdrc.org/about-us/lat... #DMD

Sam McDonald, a member of the JWMDRC Patient Registries team, recently supported a paper in the Orphanet Journal of Rare Diseases! 👏 link.springer.com/article/10.1...

Sander van Boom from LUMC explains how the #ODRL policy expression language can be used in the platform to allow patients correct permissions to access their data, at the PaLaDIn General Assembly in Rome

Joanne Lee gives an update on the Communication activities within the project at the PaLaDIn General Assembly in Rome

🚨 Big news for the #RareDisease community! The UK's DHSC launched the 2025 Action Plan for faster diagnoses, better care & treatments. Meanwhile, its French counterpart unveiled #PNMR4 to boost care, research & innovation.🏗️ ▶️UK: loom.ly/t17W4H0 ▶️FR: loom.ly/r8z1Umw

Group workshop and discussion on the PaLaDIn platform, David Allison talks about the importance of keeping the patients informed when they are providing new data

Becca Leary, Senior Project Manager for the PaLaDIn project gives an overview for the overall objectives at the General Assembly in Rome today

Graham Kay, the Technical Lead for the PaLaDIn project, gives an overview for the new patient-centric platform at the General Assembly in Rome

The @jwmdrc.bsky.social national patient registries for #FSHD and #MyotonicDystrophy are proud to support #RareDiseaseDay! Our work to help advance #neuromuscular research is not possible without pioneering patients, parents & families sharing their health data - a huge ⭐️THANK YOU⭐️ to you all! 👏🏅📝

#RareDiseaseDay | Why are patients crucial for setting funding priorities in rare disease research?🏦 Florence Guillot, Funding co-leader at #ERDERA (French National Research Agency), shares how ERDERA’s funding strategy and priorities are driving innovation in rare disease research🚀💡

Join leading experts working in #RareDisease research at our #GRD25 conference. 📅 Dates: 9-11 April 2025 💭 Share insights in person Explore the latest #genomics advances accelerating improvements in clinical care for rare disorders, globally. ⏰Secure your place by 11 March: bit.ly/3BpAe44

📢 Join us for the Rare Disease Research Conference at UCD O’Reilly Hall on 10th April. Explore rare disease research, registries, clinical trials, and lived experiences. Network with others who are passionate about rare disease! 🌟Free registration! www.eventbrite.ie/e/...

📢 One week until #RareDiseaseDay! On 28 February, we stand with the 300 million people living with a rare disease to raise awareness, promote policy change, and advance research. 🌍💙 🗓️ Stay tuned for more updates as we mark this important day! #RareDiseases

Colleagues from the JWMDRC travelled to India to meet neuromuscular colleagues with an interest in the neuromuscular disorders from Bombay Hospital, AIIMS Delhi and NIMHANS Bangalore to discuss opportunities for collaboration focussed on the Limb Girdle Muscular Dystrophies #LGMD

3 weeks to go, places are filling up! Join #GeneticsMatters for talks and hands-on science, and support #raredisease research. tinyurl.com/54vvsb3r It's free, age 12+ @rdndlab.bsky.social @rarediseasepatient.bsky.social @rarediseases.bsky.social @newcastleuni.bsky.social @medicsforrare.bsky.social

The curators' meeting was also a fantastic opportunity for our newest team members, Data Coordinators Lucy and Aleks, to learn more about the network and meet the rest of the TGDOC family. They presented registry posters, made lots of valuable connections and represented our team brilliantly! 👏🧬

Great to see so many of our global registry network colleagues at the TREAT-NMD Annual curators meeting in Milan this week! 🌍🤝 Our registries team were involved throughout, leading disease subgroup discussions, presenting our registries and making new connections with registries around the world...