Colleagues from the JWMDRC are enjoying the 17th UK Translational Research Conference for NMD in London

ICGNMD UCL_QS_CNMD

One of our PhD students, Priyanka Mehra, exploring targeting adipogenic differentiation of fibro-adipogenic precursors in DMD at the 17th UK Neuromuscular Translational Research Conference

ICGNMD UCL_QS_CNMD

Adult SMA Reach Project Manager, Sonia Segovia, gives a presentation on data collection and use from this important international study
ICGNMD UCL_QS_CNMD

For more info:
adultsmareach.co.uk

Final flash! Priyanka Mehra from Newcastle exploring targeting adipogenic differentiation of fibro-adipogenic precursors in DMD UCL_QS_CNMD

Next Newcastle’s Sonia Sergovia Simon refining data use from the important international REACH SMA study UCL_QS_CNMD

Exciting update for rare disease enthusiasts! 🌟Registration deadline for the Final EJP RD Conference extended to May 22nd, 2024! Join us on online May 27-28, 2024 for engaging sessions on RD research. Check out the agenda and secure your spot today! ℹ️ ejprarediseases.org/ejp-rd-final-c…

PaLaDIn partners met in Leiden yesterday to discuss the design of the Interactium data platform. The platform aims to provide insights to accelerate novel drug development, improve PROMS and inform health care decision-making in NMDs. Find out more: bit.ly/49o4V4O

Intense first day at the #UKNeuromuscularTranslational Research Conference at UCL learning all about 🦵🏻🧬🦠🚴‍♂️with part of the JWMDRC and Newcastle University 👨🏼‍🔬🧑🏽‍🔬👩🏻‍🔬🧑🏽‍💻 team.

The deadline to submit your abstract for #WMS2024 is fast approaching. You only have until midnight (BST) tomorrow (Wednesday, 17th April). You'll get the link to submit an abstract when you register for the Congress. Find out more and register online: buff.ly/3PZDNBR

#c4c has established the #YIS , aiming to improve collaboration, education, and networking within the network. By including the future leaders, we can further increase the capacity for the management of multinational #PaediatricClinicalTrials across Europe. conect4children.org/young-investig…

Did you know we are hosting a Symposium and networking event this autumn in Boston?

Developing Therapies in Rare Neuromuscular Disease will be a day of insightful talks on topics and networking opportunities for industry professionals: treat-nmd.org/what-we-do/tac…

🧫🧬 A new study co-led by Institut de Recerca Sant Joan de Déu and Centro Andaluz de Biología del Desarrollo uncovers the connection between mutations in the #GEMIN5 gene and #CoenzymeQ10 deficiency, opening new therapeutic pathways.

More details on the blog 👉 share4rare.org/news/study-mut…
#rarediseases #mitochondria

Today is Pompe disease awareness day. John Foxwell, who lives in Wales, shines a light on the importance of education for better diagnosis and the huge strides he's made to raise awareness. Read more: shorturl.at/hmsBL

🎉We're thrilled to announce that we've been awarded part of the Medical Research Charities Early Career Researcher Fund. This will fund seven researchers in the early stages of their career studying muscle wasting and weakening conditions.

Read more:
shorturl.at/wCOWX

Join next week’s Rare Disease Forum 2024, co-organised by EURORDIS, featuring ‌Yann Le Cam, for discussion on the EU's role in enhancing competitiveness in life sciences, fostering investment, and the benefits of a competitive agenda.

Register now ➡️ events.global-counsel.com/RareDiseasesFo…

Delighted to kick off National mitochondrial patient day Queen Square. Thanks to our great team for organising and for all patients/ families for attending. Good to see huge increase in advanced therapy trials for mito diseases UCLH UCL Brain Sciences The Lily Foundation UCL Queen Square Institute of Neurology

Would you like to start a #research project on #rarediseases on our platform? This might interest you 👇

💻 Tune in on May 9th to our #webinar , where we will delve into the details of this year's #Share4Rare 𝗖𝗮𝗹𝗹𝟰𝗣𝗿𝗼𝗷𝗲𝗰𝘁𝘀.

Registrations ✍️ share4rare.org/news/share4rar…

I am presenting this poster today at the IDMC congress in Nijmegen. This is an international collaborative study to understand the burden of patients with Myotonic Dystrophy type I. JWMDRC

If you're a person living with Duchenne muscular dystrophy or care for someone who does, help us understand patient community priorities for physio- and occupational-therapy care. You can complete the DMD-Care UK survey here: newcastle.onlinesurveys.ac.uk/parent-survey-…

📢 Call for Poster Abstracts for the TREAT-NMD 2025 Conference in Dubai! Showcase achievements, demonstrate strategies, foster collaboration. Submit now > treat-nmd.org/conference/abs… #TREATNMD #NeuromuscularDiseases #HealthcareInnovation