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.orghttps://bsky.app/profile/oxfordharrington.bsky.socialhttps://bsky.app/profile/oxfordharrington.bsky.social/post/3mvauzxzgfu2iScientists have created the largest molecular map of autism to date, identifying 1,800+ protein interactions involving 250+ autism-associated genes. The research could help identify shared pathways — and potential therapeutic targets across multiple genetic forms of autism. 🔗 buff.ly/CA8mHoK11 Sep 2026 15:29 +0000at://did:plc:7rm3zq6nutd5zzlv6eiat4dc/app.bsky.feed.post/3mvauzxzgfu2ihttps://bsky.app/profile/oxfordharrington.bsky.social/post/3muk2iwnfht2xFor generations, Bonnie Woolston’s family lived with a rare, unnamed motor neuron disease. Today, we know it as ALS4. 2025 Oxford-Harrington Rare Disease Scholar Albert La Spada, MD, PhD, is advancing RNA-based research towards a potential disease-modifying therapy. Read more: buff.ly/iPKsVmR02 Sep 2026 13:36 +0000at://did:plc:7rm3zq6nutd5zzlv6eiat4dc/app.bsky.feed.post/3muk2iwnfht2xhttps://bsky.app/profile/oxfordharrington.bsky.social/post/3mrap6v6heq26We'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 #RareDiseaseResearch22 Jul 2026 16:04 +0000at://did:plc:7rm3zq6nutd5zzlv6eiat4dc/app.bsky.feed.post/3mrap6v6heq26https://bsky.app/profile/oxfordharrington.bsky.social/post/3mqr6at3pjr2f🧬 #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/xowFgpj16 Jul 2026 11:50 +0000at://did:plc:7rm3zq6nutd5zzlv6eiat4dc/app.bsky.feed.post/3mqr6at3pjr2fhttps://bsky.app/profile/oxfordharrington.bsky.social/post/3mqoqfzxbuy2vToday 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/LQO4J9A15 Jul 2026 12:37 +0000at://did:plc:7rm3zq6nutd5zzlv6eiat4dc/app.bsky.feed.post/3mqoqfzxbuy2vhttps://bsky.app/profile/oxfordharrington.bsky.social/post/3mq5i6lscm32lWe 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 #ERDERA08 Jul 2026 15:55 +0000at://did:plc:7rm3zq6nutd5zzlv6eiat4dc/app.bsky.feed.post/3mq5i6lscm32lhttps://bsky.app/profile/oxfordharrington.bsky.social/post/3mpqn7qjfr22qGreat 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.03 Jul 2026 13:20 +0000at://did:plc:7rm3zq6nutd5zzlv6eiat4dc/app.bsky.feed.post/3mpqn7qjfr22qhttps://bsky.app/profile/oxfordharrington.bsky.social/post/3mpls7fi5uv2gWe'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 #ChildhoodCancerResearch01 Jul 2026 15:06 +0000at://did:plc:7rm3zq6nutd5zzlv6eiat4dc/app.bsky.feed.post/3mpls7fi5uv2ghttps://bsky.app/profile/oxfordharrington.bsky.social/post/3mp2dpj2zco2kAs 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/tfadvK624 Jun 2026 16:32 +0000at://did:plc:7rm3zq6nutd5zzlv6eiat4dc/app.bsky.feed.post/3mp2dpj2zco2khttps://bsky.app/profile/oxfordharrington.bsky.social/post/3mongpcrybh25At 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.uk19 Jun 2026 13:21 +0000at://did:plc:7rm3zq6nutd5zzlv6eiat4dc/app.bsky.feed.post/3mongpcrybh25https://bsky.app/profile/oxfordharrington.bsky.social/post/3mnu7m3wfgi26Stay 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/khXMpAl09 Jun 2026 12:37 +0000at://did:plc:7rm3zq6nutd5zzlv6eiat4dc/app.bsky.feed.post/3mnu7m3wfgi26https://bsky.app/profile/oxfordharrington.bsky.social/post/3mnhkxotx532uAs 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 #OxfordHarrington04 Jun 2026 11:56 +0000at://did:plc:7rm3zq6nutd5zzlv6eiat4dc/app.bsky.feed.post/3mnhkxotx532uhttps://bsky.app/profile/oxfordharrington.bsky.social/post/3mmyl4jcco72d🧬 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. 🔗 https://www.youtube.com/@genebrainbreakthrough29 May 2026 12:49 +0000at://did:plc:7rm3zq6nutd5zzlv6eiat4dc/app.bsky.feed.post/3mmyl4jcco72dhttps://bsky.app/profile/oxfordharrington.bsky.social/post/3mmralwagok26We 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. [contains quote post or other embedded content]26 May 2026 14:52 +0000at://did:plc:7rm3zq6nutd5zzlv6eiat4dc/app.bsky.feed.post/3mmralwagok26https://bsky.app/profile/oxfordharrington.bsky.social/post/3mlblu7hdpe2gWe 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 https://tinyurl.com/y6nwj5b207 May 2026 16:05 +0000at://did:plc:7rm3zq6nutd5zzlv6eiat4dc/app.bsky.feed.post/3mlblu7hdpe2ghttps://bsky.app/profile/oxfordharrington.bsky.social/post/3mkkvbbqcna26At 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.28 Apr 2026 15:23 +0000at://did:plc:7rm3zq6nutd5zzlv6eiat4dc/app.bsky.feed.post/3mkkvbbqcna26https://bsky.app/profile/oxfordharrington.bsky.social/post/3mk3praxoyv2bThis 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! https://justgiving.com/page/ploddingprofessor-curedhdds?utm_medium=FR&utm_source=CL22 Apr 2026 14:34 +0000at://did:plc:7rm3zq6nutd5zzlv6eiat4dc/app.bsky.feed.post/3mk3praxoyv2bhttps://bsky.app/profile/oxfordharrington.bsky.social/post/3mjpazs5tdp2iThis #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. #Neuroscience17 Apr 2026 15:39 +0000at://did:plc:7rm3zq6nutd5zzlv6eiat4dc/app.bsky.feed.post/3mjpazs5tdp2ihttps://bsky.app/profile/oxfordharrington.bsky.social/post/3mj5gwf64er2jAs 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.10 Apr 2026 13:36 +0000at://did:plc:7rm3zq6nutd5zzlv6eiat4dc/app.bsky.feed.post/3mj5gwf64er2jhttps://bsky.app/profile/oxfordharrington.bsky.social/post/3mijl3x6rzo2uLast 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.02 Apr 2026 15:58 +0000at://did:plc:7rm3zq6nutd5zzlv6eiat4dc/app.bsky.feed.post/3mijl3x6rzo2uhttps://bsky.app/profile/oxfordharrington.bsky.social/post/3mhvnnyrw5p2nProgress 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. #RareDisease25 Mar 2026 17:50 +0000at://did:plc:7rm3zq6nutd5zzlv6eiat4dc/app.bsky.feed.post/3mhvnnyrw5p2nhttps://bsky.app/profile/oxfordharrington.bsky.social/post/3mhgfecqlnq2rWe’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 https://tinyurl.com/4zht96ry19 Mar 2026 16:12 +0000at://did:plc:7rm3zq6nutd5zzlv6eiat4dc/app.bsky.feed.post/3mhgfecqlnq2rhttps://bsky.app/profile/oxfordharrington.bsky.social/post/3mhbdmdpt252zFriedreich’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 https://tinyurl.com/2j5v3emn17 Mar 2026 15:57 +0000at://did:plc:7rm3zq6nutd5zzlv6eiat4dc/app.bsky.feed.post/3mhbdmdpt252zhttps://bsky.app/profile/oxfordharrington.bsky.social/post/3mgpjnghuod27For 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.10 Mar 2026 13:57 +0000at://did:plc:7rm3zq6nutd5zzlv6eiat4dc/app.bsky.feed.post/3mgpjnghuod27https://bsky.app/profile/oxfordharrington.bsky.social/post/3mgczaoh6eb2zRare 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/cbxr5vdm05 Mar 2026 14:32 +0000at://did:plc:7rm3zq6nutd5zzlv6eiat4dc/app.bsky.feed.post/3mgczaoh6eb2zhttps://bsky.app/profile/oxfordharrington.bsky.social/post/3mgagnlmpvt2rClosing 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/mr3k3tu904 Mar 2026 13:54 +0000at://did:plc:7rm3zq6nutd5zzlv6eiat4dc/app.bsky.feed.post/3mgagnlmpvt2rhttps://bsky.app/profile/oxfordharrington.bsky.social/post/3mftvvn2qcs25#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 #LightUpForRare27 Feb 2026 14:22 +0000at://did:plc:7rm3zq6nutd5zzlv6eiat4dc/app.bsky.feed.post/3mftvvn2qcs25https://bsky.app/profile/oxfordharrington.bsky.social/post/3mfrbn6l2lk2xThank you to RARE Revolution Magazine® for spotlighting our work to turn scientific discoveries into real medicines for rare diseases. Read more: tinyurl.com/eyfrudcx26 Feb 2026 13:14 +0000at://did:plc:7rm3zq6nutd5zzlv6eiat4dc/app.bsky.feed.post/3mfrbn6l2lk2xhttps://bsky.app/profile/oxfordharrington.bsky.social/post/3mf532quo2l2jWith 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/mr3k3tu918 Feb 2026 12:23 +0000at://did:plc:7rm3zq6nutd5zzlv6eiat4dc/app.bsky.feed.post/3mf532quo2l2jhttps://bsky.app/profile/oxfordharrington.bsky.social/post/3meykjopnkf2xRare 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.16 Feb 2026 17:16 +0000at://did:plc:7rm3zq6nutd5zzlv6eiat4dc/app.bsky.feed.post/3meykjopnkf2x