Oxford-Harrington Rare Disease Centre
@oxfordharrington.bsky.social
Partnership of the University of Oxford and Harrington Discovery Institute. Combining expertise in discovery science and therapeutics development to accelerate cures for rare diseases.
Visit us at: www.oxfordharrington.org
Visit us at: www.oxfordharrington.org
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Transforming Rare Disease Treatment | The Oxford-Harrington Rare Disease Centre
YouTube video by Oxford-Harrington Rare Disease Centre
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🧬 95% of rare diseases have no treatment. This Rare Disease Day, we’re working to change that by accelerating breakthroughs—40 new treatments by 2034—through world-class research & drug development.
Learn more: tinyurl.com/2ab5zecf
#RareDiseaseDay #CareForRare www.youtube.com/watch?v=sKEf...
Learn more: tinyurl.com/2ab5zecf
#RareDiseaseDay #CareForRare www.youtube.com/watch?v=sKEf...
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
Photos: Jeremy Peters
#RAREsummit25
November 18, 2025 at 4:15 PM
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
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."
He said: "It was valuable to highlight the essential role of academic drug development in addressing rare diseases and the importance of regulatory innovation."
November 14, 2025 at 4:26 PM
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."
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.
November 7, 2025 at 4:07 PM
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
A more adaptive, evidence-based framework will help researchers and clinicians deliver life-changing therapies faster.
Read more: tinyurl.com/msurx5ju
New drugs to be approved faster so patients don’t wait years
The medicines watchdog is changing how it oversees research of 7,000 rare diseases, which collectively affect about one in 17 Britons
tinyurl.com
November 3, 2025 at 4:22 PM
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
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
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
Events Calendar for Harrington Discovery Institute | Harrington Discovery Institute at University Hospitals
Upcoming and past events for Harrington Discovery Institute showcasing how grant funding accelerates science into medicines for diseases, social impact fundraising
www.harringtondiscovery.org
October 31, 2025 at 10:40 AM
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
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
📅 11–12 Nov | London & online
🔗 bit.ly/4pPcJpd
#FTPharma #Biotech
October 27, 2025 at 1:59 PM
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
📅 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
Register now: www.harringtondiscovery.org/events
#OxfordHarrington #RareDisease
Events Calendar for Harrington Discovery Institute | Harrington Discovery Institute at University Hospitals
Upcoming and past events for Harrington Discovery Institute showcasing how grant funding accelerates science into medicines for diseases, social impact fundraising
www.harringtondiscovery.org
October 24, 2025 at 12:14 PM
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
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
🔗 Register: tinyurl.com/4xe26ppm #AccelerateRare
October 10, 2025 at 4:23 PM
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
🔗 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
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
How Patient Stories Fuel Ataxia Research
On International Ataxia Awareness Day (25 September), the spotlight turns to the progress being made to treat rare neurological conditions. For Autosomal Recessive Spastic Ataxia of
tinyurl.com
September 25, 2025 at 11:24 AM
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
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 19, 2025 at 12:42 PM
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.
September 17, 2025 at 6:03 PM
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...
📅 19 Sept | 11:45–12:45
📍 IDRM, Old Road Campus
🔗 Register for free: talks.ox.ac.uk/talks/id/afb...
September 16, 2025 at 3:57 PM
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...
📅 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
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
September 7, 2025 at 12:00 PM
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
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
Read more in this article by Jason Mast: shorturl.at/zBQmI
As Sarepta Therapeutics fights for Duchenne therapy, a group of patients gets left behind
After deaths and FDA setbacks, Sarepta abandoned LGMD gene therapy, leaving families desperate and patients without long-promised treatments.
shorturl.at
September 3, 2025 at 4:15 PM
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
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
DSUK Conference - Professional Day
Biocodex, Encoded Therapeutics, Harmony Biosciences, Jazz Pharmaceuticals and Stoke Therapeutics have all provided sponsorship funding to DSUK in support of the Biennial Professional Day Conference.…
bit.ly
August 14, 2025 at 4:07 PM
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
Read more: tinyurl.com/d37tvuxb
July 24, 2025 at 12:10 PM
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
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=...
The OHC newsletter contains updates on our latest projects, funding announcements, events, and initiatives.
Sign up now: oxfordharrington.us17.list-manage.com/subscribe?u=...
July 16, 2025 at 12:31 PM
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=...
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
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
How genetic testing helped Albert fight a rare neonatal epilepsy
One-year-old Albert lives with Early Infantile Developmental and Epileptic Encephalopathy, EIDEE, a rare form of neonatal epilepsy. Thanks to advances in genetic testing by the Exeter Genomics
shorturl.at
July 3, 2025 at 3:32 PM
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
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
Read more about the project:🔗 lnkd.in/gntnHenF
June 30, 2025 at 1:18 PM
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
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
Link to the full article: shorturl.at/pvyoC
Should wealthy people be allowed to buy their way on to drug trials?
It was a handwritten birthday party invitation that finally made Mel Dixon realise something was wrong.
shorturl.at
June 25, 2025 at 3:34 PM
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
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.
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.
June 20, 2025 at 10:55 AM
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.
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
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
June 19, 2025 at 12:33 PM
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
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 🔗
Open to Oxford researchers.
Deadline: 18 June 2025
More info: shorturl.at/i5BwY 🔗
June 4, 2025 at 12:29 PM
📢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 🔗
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
#CysticFibrosis #RareDisease
May 28, 2025 at 1:33 PM
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
#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
Read more: shorturl.at/ri7QU
May 27, 2025 at 12:02 PM
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
Read more: shorturl.at/ri7QU