We're delighted to be included on Raremap, a new guide to the UK's rare disease research ecosystem developed by @lifearc.bsky.social and @rdrukhub.bsky.social. Explore: 🔗 : buff.ly/yQx6pYT #RareDiseases #RareDiseaseResearch

🧬 #RareDiseaseNews Newborn screening for #SpinalMuscularAtrophy (#SMA) will begin rolling out across England from October 2026, helping babies access diagnosis and treatment before symptoms appear. A significant step forward for early diagnosis and #raredisease care. buff.ly/xowFgpj

Today is #CASKAwarenessDay. When Sarah was diagnosed with a rare CASK-related disorder, her family was told there were no disease-modifying treatments. Her mother Laura founded @caskresearch.bsky.social to bring awareness and hope to the CASK community. buff.ly/LQO4J9A

We welcome the UK government's announcement that the National Institute for Health and Care Research (NIHR) has joined the European Rare Diseases Research Alliance (ERDERA). Read more: buff.ly/mDuFotc #RareDisease #RareDiseaseResearch #ERDERA

Great to be part of #GERS2026 last week. At the Genomics England Research Summit, OHC Genomics Lead, Stephan Sanders shared how the MRC CoRE in Therapeutic Genomics and partners are building new pathways to accelerate genetic medicines.

We're pleased to welcome Professor Isidro Cortés Ciriano isidrolauscher.bsky.social as the first Little Princess Trust Professor of Paediatric Oncology at @ox.ac.uk, @paediatrics.ox.ac.uk and Cancer Theme Lead at the OHC! Read more: buff.ly/Sg5mWou #RareDisease #ChildhoodCancerResearch

As a founding partner, we welcome the announcement that the Rare Therapies Launch Pad (#RTLP) will become part of @lifearc.bsky.social in a bid to accelerate the development and access of individualised medicines for people living with rare diseases. Read more🔗: buff.ly/tfadvK6

At the Rare Diseases Network Event at Brunel University London, members of the FA Alliance at Oxford shared updates from six philanthropically funded projects focused on developing new therapeutic approaches for #FriedreichsAtaxia. @ox.ac.uk @kavli.ox.ac.uk

Stay up to date with the latest news from the Oxford-Harrington Rare Disease Centre (OHC). Our quarterly newsletter includes science news, programme updates, event announcements, and highlights from across the OHC community. Subscribe to our mailing list: buff.ly/khXMpAl

As highlighted by Brandon Carrus in his discussion with the Chair of the Oxford-Harrington Advisory Council, David Cameron, scientific breakthroughs only change lives when they reach patients. 🔗 buff.ly/nLZmtdI #RareDisease #OxfordHarrington

🧬 We're pleased to share Genes, Brains, and Breakthroughs, a new educational animation series helping make topics like #genetics, #neurodevelopmentaldisorders, #rarediseases, and emerging therapies more accessible. 🔗 www.youtube.com/@genebrainbr...

We welcome the @mhragovuk.bsky.social landmark plans to accelerate the development and approval of treatments for rare diseases. For the 3.5M people in the UK living with a rare condition, many without an approved treatment, faster pathways for innovative therapies could be transformative.

University of Oxford@ox.ac.uk · 2mo ago

Rare diseases affect 3.5 million people in the UK, yet most still have no approved treatment. Prof Matthew Wood has welcomed new government plans designed to speed up access to cutting-edge therapies for patients and families living with rare conditions ⬇️ https://bit.ly/4dJdQBB

We are proud to share a transformational $10M investment from Dee and Jimmy Haslam to accelerate research and drug development for chronic lymphocytic leukaemia (CLL) and other rare blood cancers through the Oxford-Harrington Rare Disease Centre. Read more: tinyurl.com/y6nwj5b2

Dee and Jimmy Haslam Make $10 Million Transformational Investment in Blood Cancer Research and Treatments at the Oxford-Harrington Rare Disease Centre

The Oxford-Harrington Rare Disease Centre has received $10 million in transformational support from Dee and Jimmy Haslam to fuel research and drug development for chronic lymphocytic leukaemia (CLL)

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At the @rdrukhub.bsky.social 3rd Annual Conference, the theme “The Power of Collaboration,” emphasised how partnerships between organisations - @lifearc.bsky.social, @geneticallianceuk.bsky.social, patients and carers, can build a stronger rare disease ecosystem for #RareDisease drug development.

This weekend, Oxford’s “Plodding Professor” takes on the London Marathon for Cure DHDDS to help raise awareness for an ultra‑rare neurological disorder, with wider research implications for conditions such as Parkinson’s and Alzheimer’s. Good luck, Prof Edwards! justgiving.com/page/ploddin...

Plodding Professor for Cure DHDDS (@ploddingprofessor)

Help James Edwards raise money to support Cure DHDDS

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This #AutismAwarenessMonth, we’re highlighting research at OHC advancing new approaches to neurodevelopmental disorders. Work led by OHC Co-Director Matthew Anderson is helping define the biological mechanisms underlying autism to enable more precise, targeted therapeutic strategies. #Neuroscience

As an Oxford-Harrington Rare Disease Scholar, Dr. Jacquelyn Bower is developing an AAV gene therapy for uveal melanoma, a rare eye cancer that often leads to blindness. Her approach targets a key mutation driving tumor survival, with potential to extend life and preserve vision.

Last week, at the LifeArc Centre for Rare Respiratory Diseases event, our Director and CSO, Prof Matthew Wood, joined Volker Straub and Jason Mellad, in a panel chaired by Samantha Walker, to discuss how to accelerate the path from rare disease drug discovery to patient impact.

Progress in rare disease research takes more than innovation—it takes shared urgency. OHC Scholar Prof Carlo Rinaldi is developing a novel RNA-based therapy for SBMA while working closely with a team that helps drive ideas forward and accelerate impact. #RareDisease

We’re delighted that OHC Co-Director Matthew Anderson has been appointed Visiting Professor at @ox.ac.uk A leader in neuroscience, genetics & therapeutics, he’ll help drive collaboration, mentorship, and innovation in rare disease research. 🔗Read more: tinyurl.com/4zht96ry

Matthew Anderson, MD, PhD, OHC Co-Director, Appointed Visiting Professor at the University of Oxford

The University of Oxford has appointed Matthew P. Anderson, MD, PhD, as a Visiting Professor in its Department of Paediatrics—an honor that reflects both his scientific accomplishments and his

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Friedreich’s ataxia research is entering a new era. As @BioCentury highlights, the field is shifting toward targeting the root cause of disease — restoring FXN. At the OHC, we’re supporting Oxford-led research through the FA Alliance Innovation Fund. 🔗 Read more: tinyurl.com/2j5v3emn

Rare disease spotlight: Friedreich ataxia moves beyond mitochondrial bandages

Friedreich ataxia is moving into a mechanistic second act, as biotechs pivot from stabilizing the mitochondrial damage caused by frataxin loss to developin...

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For Rare Disease Day 2026, Genetic Alliance UK brought together patients and stakeholders, including Rare Disease Research UK, Oxford-Harrington Rare Disease Centre, LifeArc, Medical Research Council and many others, at a UK parliamentary reception to raise awareness.

Rare diseases affect an estimated 500 million people worldwide (1 in 17). Yet around 95% of 10,000+ known rare diseases have no approved treatment. In Beyond Biotech by Labiotech.eu, Professor Matthew Wood discusses scalable antisense and gene editing approaches. 🔗 Listen: tinyurl.com/cbxr5vdm

Closing Soon: Oxford-Harrington Rare Disease Scholar Award 2026 Independent academic researchers in the UK, US & Canada can apply for £100K/$100K over two years, plus dedicated drug development support for rare diseases. Deadline: 9 March 2026. 🔗 : tinyurl.com/mr3k3tu9

#RareDiseaseDay, marked each year on the last day of February, shines a light on the 500 million people worldwide living with a rare condition. To mark the day, the OHC illuminated @magdalenoxford.bsky.social in the colours of @rarediseaseday.bsky.social as part of the global #LightUpForRare

Thank you to RARE Revolution Magazine® for spotlighting our work to turn scientific discoveries into real medicines for rare diseases. Read more: tinyurl.com/eyfrudcx

With less than one month to go until the 9 March deadline, this is your reminder to apply for the Oxford-Harrington Rare Disease Scholar Award 2026. £100K/$100K in funding + drug development support for academic researchers in the UK, US & Canada. 🔗 tinyurl.com/mr3k3tu9

Rare diseases affect an estimated 500M people worldwide, yet approximately 95% still have no approved treatment. As we look ahead to Rare Disease Day on February 28th, we stand with the rare disease community and reaffirm our commitment to turning breakthrough science into life-changing medicine.

The Oxford-Harrington Rare Disease Centre has awarded philanthropic funding of £500,000 through the FA Alliance Innovation Fund to support five Oxford researchers advancing new therapeutic approaches for Friedreich’s Ataxia, a devastating rare disease with no cure. Read more: tinyurl.com/4srwmjza

The Oxford-Harrington Rare Disease Centre Advances Discovery of Therapies for Friedreich’s Ataxia (FA)

The Oxford-Harrington Rare Disease Centre (‘OHC’) dedicated to creating new medicines for rare diseases, today announced that the FA Alliance Innovation Fund has awarded funding to five researchers

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The next VITALS session will feature Matthew Anderson, MD, PhD, a global leader in rare disease innovation and translational science. He is the co-director of the Oxford-Harrington Rare Disease Centre, with groundbreaking work spanning academia and industry. Register: tinyurl.com/f534nvw4