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

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...

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

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

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 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

The Oxford-Harrington Rare Disease Centre invites proposals for its 2026 Rare Disease Scholar Award, supporting the development of innovative therapies for rare diseases. Submissions close on March 9, 2026. Learn more: tinyurl.com/3ynf6xt7

In a recent episode of RARECast, Matthew Wood, Director and CSO of the OHC, discusses the challenges of rare disease drug development, the resources the OHC brings to address them, and the mechanisms established to help accelerate therapeutic development. Listen now: art19.com/shows/rareca...

In a recent episode of the In Vivo podcast, Matthew Wood discusses why rare disease research and drug development are at a pivotal moment and what must change to unlock progress for the ~500 million people impacted by rare diseases. 🎧Listen now: shorturl.at/y88MR #RareDiseases #DrugDevelopment

As the year draws to a close, we are grateful to our Scholars, partners and Advisory Council members for their continued commitment to advancing treatments and cures for rare diseases.

Last week, the Oxford-Harrington Rare Disease Centre convened its Advisory Council in Oxford to reflect on a year of progress and to set priorities for 2026. Read more: tinyurl.com/5c2kwcny

A special seminar on the human impact of ultra-rare diseases—and the growing momentum behind Cure DHDDS —takes place next Tuesday, 2 Dec (2:30–3:30pm) at the Botnar Research Centre, Oxford. 🔹 Melanie Dixon (CureDHDDS) 🔹 Prof. Emyr Lloyd-Evans (Cardiff University) 🔗 talks.ox.ac.uk/talks/id/b90...

Today, we are pleased to announce the 10 outstanding scientists selected for the 2025 Rare Disease Scholar Award, who will drive scientific breakthroughs to transform treatment options for the 500M people worldwide affected by rare diseases. Read more: tinyurl.com/49baysns

Earlier in November, the OHC team joined the programme and conversations at the CamRare Summit 2025, exploring how scientific advances, policy shifts and new models of collaboration could shape the next decade of rare disease research and care. Photos: Jeremy Peters #RAREsummit25

Earlier this week, Prof Matthew Wood, OHC Director, spoke at the FT Live Global Pharma & Biotech Summit. He said: "It was valuable to highlight the essential role of academic drug development in addressing rare diseases and the importance of regulatory innovation."

Yesterday, we heard from Professor Carlo Rinaldi, Neurological Disorders Theme Lead at the Oxford-Harrington Rare Disease Centre, who spoke at the Cambridge Rare Disease Network (CamRARE) Rare Summit 2025 about the next decade of rare disease innovation.

MHRA reforms mark a vital step toward faster, safer innovation for the 500M people worldwide living with rare diseases. A more adaptive, evidence-based framework will help researchers and clinicians deliver life-changing therapies faster. Read more: tinyurl.com/msurx5ju

Every breakthrough starts with a bold idea—and the support to make it real. Join the Oxford-Harrington Rare Disease Centre webinar to hear how scientists & philanthropists are working together to advance cures for the world’s rarest diseases. 🔗 www.harringtondiscovery.org/events

Professor Matthew Wood, Director of the Oxford-Harrington Centre, joins the FT Live Global Pharma and Biotech Summit to explore the future of healthcare innovation. 📅 11–12 Nov | London & online 🔗 bit.ly/4pPcJpd #FTPharma #Biotech

Join Oxford-Harrington Scholars Angela Russell and Michael Pacold as they share advances in Duchenne muscular dystrophy and HDPL Encephalopathy in our upcoming webinars on November 5th and 13th. Register now: www.harringtondiscovery.org/events #OxfordHarrington #RareDisease

On 14 Oct at the Francis Crick Institute, the #RareSummit2025 will unite leaders in science, healthcare & policy to accelerate diagnosis & treatment for rare diseases. 🔗 Register: tinyurl.com/4xe26ppm #AccelerateRare

On #InternationalAtaxiaAwarenessDay, we spotlight progress in ARSACS research. In Oxford, Professor Esther Becker is using patient-derived cerebellar organoids to uncover why brain cells are vulnerable and to open new paths toward treatment. 🔗 tinyurl.com/ye6k9cx4 #RareDisease #AtaxiaAwareness

Today, OHC Co-Director Dr Matthew Anderson gave a talk hosted by Prof Carlo Rinaldi, at the Institute of Developmental and Regenerative Medicine - University of Oxford, on how autism genetics provides a window into the neuronal circuits that regulate human behaviour.

September is #ChildhoodCancerAwarenessMonth, a reminder of the urgent need for new treatments.

Oxford researchers are invited to a seminar with Dr Matthew Anderson on "Neural Circuit Targets of Behavioral Medicines: Insights Through Autism Spectrum Disorder Genetics." 📅 19 Sept | 11:45–12:45 📍 IDRM, Old Road Campus 🔗 Register for free: talks.ox.ac.uk/talks/id/afb...

On #DuchenneAwarenessDay, we celebrate the power of patient advocacy. When Alex Johnson OBE and Emily Reuben OBE founded Duchenne UK, their shared mission was clear: to end Duchenne muscular dystrophy. In just over a decade, their work has transformed care across the UK. 🔗 tinyurl.com/ynz7anm5

As Sarepta's gene therapy clinical trials for limb-girdle muscular dystrophy (LGMD) have been placed on hold, many patient families have been left without alternative treatment options. Read more in this article by Jason Mast: shorturl.at/zBQmI

On 14 Nov 2025, Dravet Syndrome UK will host the #DSUKConference2025 in London St Pancras & online 🩺 The UK’s leading Dravet-focused scientific meeting will share research and clinical updates for healthcare professionals. Agenda & booking: bit.ly/DSUKProfConf25

Professor Carlo Rinaldi, one of ten 2025 Oxford-Harrington Rare Disease Scholar Award recipients, is developing a novel RNA-based therapeutic strategy for Spinal and Bulbar Muscular Atrophy (SBMA), a rare and currently untreatable neuromuscular disorder. Read more: tinyurl.com/d37tvuxb

To stay up to date with news from the OHC and the wider rare disease community, sign up for our quarterly newsletter. The OHC newsletter contains updates on our latest projects, funding announcements, events, and initiatives. Sign up now: oxfordharrington.us17.list-manage.com/subscribe?u=...

At just 4 weeks old, Albert was diagnosed with a rare neonatal epilepsy, thanks to rapid genetic testing. His mum shares how a precise diagnosis gave Albert a fighting chance, and why more progress is still needed. Read more: shorturl.at/pKH6o Support Albert: shorturl.at/rAI6v #RareDisease

Professor Michele Jacob, one of ten 2024 Oxford-Harrington Rare Disease Scholars, is developing the first targeted treatment for CTNNB1 syndrome, a rare genetic disorder with no current therapies. Read more about the project:🔗 lnkd.in/gntnHenF

Despite promising scientific advances into new therapies, many rare and ultra-rare diseases remain without treatments due to lack of funding for clinical development and trials. Link to the full article: shorturl.at/pvyoC

We’re delighted to announce that Dr Antonio García Guerra has received the first FA Alliance Catalyst Fund award. His project will develop a non-viral gene editing platform to correct the frataxin gene in cardiac muscle cells, advancing urgently needed therapeutic strategies for FA.

Are breakthrough therapies for ultra-rare diseases within reach? At the 2025 #PrixGalienUKForum, OHC Director Prof Matthew Wood joined global leaders to discuss the future of individualised medicine. Watch the panel chaired by Dame June Raine: lnkd.in/dP8_zbAE

📢Applications are still open for the FA Alliance Innovation Fund. Up to £100k available for bold, high-impact R&D projects in Friedreich’s Ataxia. Open to Oxford researchers. Deadline: 18 June 2025 More info: shorturl.at/i5BwY 🔗

Cystic fibrosis affects 105,000 people across 94 countries and is one of the most common life-threatening rare diseases worldwide. An OHC research project is developing an innovative approach to treat this genetic disease and improve patient outcomes. #CysticFibrosis #RareDisease

At last week’s Harrington Discovery Institute Scientific Symposium, OHC Advisory Council Chair, Former UK Prime Minister David Cameron, joined global scientific leaders to spotlight progress in drug development for a variety of conditions, including rare diseases. Read more: shorturl.at/ri7QU