JWMDRC

John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University #dmd #sma #lgmd #fshd #dm1 #gne #col6 #cnm

[ERDERA @ERDERA_org]

What is ERDERA, and why does it matter? 🤔
30 million people in Europe live with a rare disease. Yet most still lack a diagnosis or treatment. So what is ERDERA doing about it?
Watch the short video to find out: https://t.co/nuo3bWBysf 🔁

#ERDERA #RareDiseases

[ERDERA @ERDERA_org]

🌍 At a pivotal moment for Europe, ERDERA gathered NMGs, EU stakeholders & global partners in Sofia with one goal: stronger alignment to accelerate research & impact.
🎥 Watch the insights: https://t.co/GKbQXeJsQu

[IRDiRC @irdirc]

📣 Sign up for #RDSAConnect today!

Created by Rare Diseases South Africa, this patient-first registry helps ensure rare disease communities are seen, heard, and counted.

👉Join here: https://t.co/1WCJKvUDAy

#RareRegistry #RareDiseaseAwareness

[TREAT-NMD® @TREAT_NMD]

To mark our 20th anniversary, we announce The Past and Future of Translational Research in Neuromuscular Disease May 2027.
Building on our 2026 conference, this event reflects 20 years of progress and the future we will shape together. Register your interest to attend below.

[EURORDIS-Rare Diseases Europe @eurordis]

⏳ Less than 1 week until #ECRD2026!

Session 1 of the Preparing Reimbursement Decisions track will explore the first 18 months of HTAR implementation, including JCAs, JSCs & orphan products. Join us online: https://t.co/dvdfJAtTM7

[TREAT-NMD® @TREAT_NMD]

We're partnering with Muscular Dystrophy UK, to host our 3rd Becker Education & Engagement Day in Manchester.

If you’re living with BMD or care for someone with BMD, we’d love to see you there.
Register your interest below - (each attendee to register separately):

[JWMDRC @jwmdrc]

Volker and Meredith joined the 2nd European LGMD2I/R9 conference in Denmark this weekend, meeting people with #LGMD and families from 21+ countries.

Volker spoke on standards of care and Meredith on physio and rehab. Thanks to Kelly Brazzo and Cure LGMD2I for the invitation.

[TREAT-NMD® @TREAT_NMD]

The TREAT-NMD team are representing Project PaLaDIn at the European LGMD 2I/R9 Patient and Family Conference, providing key updates on the development of the Interactium and sharing how it will enable patients to connect, learn and make more informed decisions about their care.

[JWMDRC @jwmdrc]

Today we celebrate #ClinicalTrialsDay and the incredible #ClinicalResearch community whose work strengthens our ‘Clinical Care & Diagnostics’ and ‘Clinical Research’ core pillars — and makes a real difference for patients worldwide

[ACRP - Assoc. of Clinical Research Profession @ACRPDC]

May 20 is when the #ClinicalResearch community comes together to honor the progress made through #ClinicalTrials & the people who make it possible. https://t.co/cyX1jyk0bJ #CTD2026 #ResearchRising

#ClinicalTrialsDay Sponsors: @HCAhealthcare @sanofi @TempusAI @thermofisher @Merck

[LifeArc @lifearc1]

Health data is key to unlocking rare disease treatments, but fragmentation is slowing progress.

At #LATSS2026, Melanie Ivarsson set out a vision for a UK health data service to support research and improve access to clinical trials.

Stay tuned for recordings from the summit.

[IRDiRC @irdirc]

📣 Join ERDERA’s Diagnostic Research Workstream webinar with Holm Graessner!

Learn how data sharing, advanced diagnostic pipelines & multi-omics innovation are accelerating rare disease diagnosis.

👉 Register: https://t.co/HioDY1pcLT

#RareDiseases #ERDERA

[LGMD Awareness Foundation, Inc @LgmdAwareness]

LGMD patients and their family members are now able to access peer support via the internet. No longer do individuals diagnosed with LGMD, family members and/or caregivers have to feel alone in dealing with this rare disease. https://t.co/xxQynZTfrN

[LifeArc @lifearc1]

🔬 We're looking for senior scientific leaders to join us.

Two leadership opportunities are now open to help shape impactful drug discovery programmes tackling urgent unmet medical needs.

Learn more and apply here: https://t.co/gSuUIuZLr6

[IRDiRC @irdirc]

⏳ 6 days left to apply!

The IRDiRC TSC is looking for experts in rare diseases, digital health, modelling, and regulatory science. Don’t miss your chance to shape global collaboration.

📅 Deadline: 11 May 2026
📩 [email protected]
💡https://t.co/tcxbRloURk

[JWMDRC @jwmdrc]

The latest copy of #LGMD News is out now and contains an article by Brad Williams from @JainFoundation and our own Marianela Schiava on "LGMD & Medical Misinterpretation".

Click here to read: https://t.co/UQdLG3njwZ

@speakfoundation

[JWMDRC @jwmdrc]

Interested in uncovering why muscles weaken and fail in rare genetic diseases—and how this knowledge could help develop future therapies?

We have a great new PhD opportunity for anyone interested in muscle biology, mitochondria, & rare disease research!

https://t.co/HDuH3cAJ1c

[EURORDIS-Rare Diseases Europe @eurordis]

📣 New @irdirc publication on multi-omics!

How can integrating biological #data improve rare disease diagnosis and treatment? This new paper explores its potential - and the need for scalable, equitable implementation.

Read more 👇

[IRDiRC @irdirc]

📣 Global Nursing Education for Rare Diseases Position Paper Released

The GNNRD position paper outlines actions to strengthen nursing education & improve care worldwide.👩‍⚕️🩺🌍

👉 Read now: https://t.co/d4MeY3WQwA

#UndiagnosedDisease #Nurses #Education #GNNRD

[JWMDRC @jwmdrc]

The Duchenne Hub held a successful workshop for staff involved in UK DMD trials, discussing future trials, gene therapies, transition to adult services, and a “challenging conversations” session led by Maguire Communication & Leadership Academy

More info: https://t.co/5W1biN1QI1