Our recently launched Health Data Strategy sets out four goals to unlock the potential of high-quality data to transform the lives of people living with rare disease.
Read more here: https://t.ly/59v-G
In many regions, the basic ability to test for bacteria is limited or absent.
That's why we're a part of Global One Health Diagnostics Access Compact (GO‑Dx) – a collective commitment of over 20 organisations to strengthen access to diagnostics across global health systems.
Professor Chris McDermott and Dr Liam Knox (@sheffielduni.bsky.social) are developing a new software to spot MND symptom patterns in patient records and flag potential referrals.
We’re supporting this project via our Primer Fund to improve diagnosis of MND in primary care: https://t.ly/wfHNU
CognoMND is a simple digital tool that could allow people with motor neuron disease to be assessed for cognitive and behavioural changes in their own time, from the comfort of their homes.
This could support earlier detection of changes and better care management.
🔗Learn more: t.ly/m_T4O
Could a new drug help prevent progression of a rare and severely disabling genetic disease?
We're funding the STOPFOP trial, testing the use of saracatinib for people with fibrodysplasia ossificans progressiva (FOP). Learn more: http://bit.ly/43Hc13U
@ox.ac.uk @amsterdamumc.bsky.social
Individually rare. Collectively common. The @newstatesman1913.bsky.social special rare disease report explores what more can be done to deliver faster diagnoses and better treatments.
👉 Read now: https://t.ly/EyekY
We’ve partnered with HMBio and Southern Research to humanize an antibody at the heart of a potential new treatment to calm overactive immune cells in a targeted way, supporting its path towards the clinic.
How did we get thousands of commuters thinking about rare diseases?
• We met people where they are: King's Cross
• Led with a bold, contextualised statistic
• Made the message human
• Gave people something to do and something they'd keep
And yes, the life‑sized zebras helped.
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: https://t.ly/umN1O
Diagnostics are critical to early detection, effective treatment and responsible antibiotic use.
That’s why we’ve joined the Global One Health Diagnostics Access Compact (GO‑Dx), launched today at the One Health Summit – a shared commitment to improving diagnostics worldwide.
What happens to lab equipment when a facility closes?
After closing our Edinburgh labs, we donated specialist equipment to organisations in Nigeria, Zimbabwe and South Africa to strengthen research infrastructure. Other equipment found new homes locally.
Families aren't waiting for change – they're driving it. In the latest @newstatesman1913.bsky.social podcast episode, our CEO and parent-advocate, Terry Pirovolakis, explored how lived experience is reshaping rare disease research.
🎧 Listen now: https://t.ly/m1wRC
How can we give children with cancer hope for longer, healthier lives?
Our Paediatric Therapeutic Development Workshops bring experts together to focus research, prioritise drug targets and shape future trials for childhood cancers with unmet needs.
Learn more: t.ly/umN1O
⚡Good news for #GAMRIC2026: the deadline for abstract submissions has been extended to 10 April 2026! We look forward to receiving your submissions!
https://ow.ly/Vtx050YzIPo
CARB-X, LifeArc, Global Antibiotic R&D Partnership (GARDP) #IDSky #clinmicro
Professor @majidhafezparast.bsky.social and his team at the University of Sussex are validating a panel of non-coding RNAs and exploring the use of AI to potentially help diagnose MND.
We’re supporting this project via our Primer Fund to improve diagnosis of MND in primary care: https://t.ly/KyRR4
When Casey was born with a gap in his food pipe, he underwent major surgeries and needed a feeding tube. New research from @greatormondst.bsky.social and UCL, part-funded by us, points to a future of personalised regenerative treatments for children like Casey.
Read more: https://t.ly/qRH02
"Progress will rely on action as much as innovation."
With dementia set to reach 150 million cases by 2050, we must rethink financing, data and public awareness to reshape the future of brain health.
Find out how in our recent blog: https://t.ly/p1e3F
Through the MND Insights Group, we hope to create a future where research reflects what truly matters to patients and families.
"Follow what you're genuinely passionate about; stay curious and don’t be afraid to ask questions!"
Read on for more advice and reflections around pursuing a career in science from some of our industrial placement students at LifeArc.
🔗 https://t.ly/MEtJy
Rhabdoid tumours are rare cancers with limited treatment options. Our Paediatric Therapeutic Development Workshop identified 6 potential drug targets as priority and the drugs that could work best against them.
🔗Findings now published in British Journal of Cancer: https://t.ly/umN1O
Harry was diagnosed with Ewing sarcoma at age 6. After surgery, chemotherapy and proton-beam therapy, he faced a long recovery. Children like Harry need kinder, more effective cancer treatments.
Through C-Further, we're proud to be supporting a new treatment for Ewing sarcoma.
A years-long diagnostic odyssey is a reality facing many people living with a rare disease.
This special @newstatesman1913.bsky.social Spotlight podcast episode explores why. 🎧Listen now: t.ly/flE_n
At LifeArc, patients are at the heart of everything we do. During this fireside chat, our CEO shares insights from patients that have shaped her perspective, explaining why we must innovate faster and act with a sense of urgency.
Watch the full conversation: https://t.ly/ITQyq
How can we get effective treatments to rare disease patients faster?
Read perspectives from 5 industry experts in the @newstatesman1913.bsky.social’s rare disease report: https://t.ly/EyekY
How can the UK better support innovation in #RareConditions research? A new report from @geneticallianceuk.bsky.social and the @lifearc.bsky.social Translational Centres Hub explores novel trial designs, AI and multimodal data – and calls for renewal of the UK Rare Diseases Framework. bit.ly/40xbH6b
Launched to a positive reception in parliament yesterday, a new report – More trials, better data, faster access – outlines policy changes that would have huge impact for people with rare diseases.
👉 Read the report now: t.ly/LJRi4
More trials, better data, faster access.
We worked with Genetic Alliance UK to develop a new report from the LifeArc Translational Centres for Rare Disease Hub, setting out how smarter policy can accelerate rare disease research.
Read more 👉 https://t.ly/LJRi4
We're supporting the DefINe trial, testing the use of deferiprone to treat people with neuroferritinopathy, a disorder which leads to the gradual buildup of iron in the brain and ultimately the death of brain cells.
@cam.ac.uk
Learn more: https://t.ly/HEFgY
Curiosity is where science starts.
For #BritishScienceWeek, we’re sharing the stories of our placement students and degree apprentices – what inspires them, what they’re learning and their advice for future scientists.
🔗 Read now: t.ly/wWj3G
C‑Further has announced its first two projects, developing treatments for aggressive cancers in children and young people: https://t.ly/fztCX
- An immunotherapy for multiple hard-to-treat cancers
- A small molecule therapy for Ewing sarcoma
@crhorizons.bsky.social @goshcharity.bsky.social
