Congratulations to Ronjon and Jack on their fundraising efforts for Cure Leukaemia. Chakraverty Lab RDM Oxford MRC WIMM works on T cell immunotherapies for leukaemias and other #rarecancers

July 11 is our 1st Northeast Ohio Liver Alliance rare disease webinar. OPEN TO THE PUBLIC 🩺 Dr. Fred Askari Michigan Hepatology Wilson disease 🩺 Naim Alkhouri Arizona Liver Health PFIC Alice Williams Wilson Disease Assoc & Emily Ventura PFICNETWORK pt/caregiver journey #LiverTwitter liveralliance.org/events/liver-q…

Congratulations! Nicky Whiffin leads the Computational Rare Disease Genomics group in Oxford, which drives the understanding of genetic variation in #raredisease and is key to developing new therapies for patients

Thank you for the visit and for the excellent and productive discussions on how we will work together to deliver new therapies for #Friedreichsataxia

Today we are in London at the Inaugural Symposium of the UK Platform for Nucleic Acid Therapies UK Platform for Nucleic Acid Therapies- UPNAT Rare Disease Research UK which set to be an invaluable resource for the #raredisease community. Introduction by Dr Haiyan Zhou the Director of UPNAT

A powerful example in Nature Medicine of how whole genome sequencing can improve clinical care in paediatric oncology and inform individualised medicine for children with cancer Ångus Hodder Sarah Leiter Jack Bartram Aditi Vedi and teams CRUK Cambridge Centre: Paediatric Cancer 🎗️ NHS Genomic Medicine Service

Carlo Rinaldi @the_r_lab Department of Paediatrics Oxford_IDRM leads our Neurological Disorders Theme and works at the exciting interface of research and industry, collaborating with companies to deliver new therapies for #RareDiseases

Today we’re at #GERS2024 the Genomics England Research Summit. An inspiring and informative event featuring patients, funders, clinicians and scientists, with engaging and topical panel discussions for all stakeholders in genomics and #RareDisease

A hugely important discovery in neurodevelopmental disorders from the Computational #RareDisease Genomics group in Oxford. Offering hope for many thousands of people with mutations in the gene RNU4-2 Yuyang Chen Nicky Whiffin Unique Genomics England

Great meeting at Westminster Health Forum WHF Events, Priorities for rare disease diagnosis, care & treatment in England, with talks from patients, researchers, companies and funders Timothy Barrett Adam Galloway Medics4RareDiseases Charlotte Chapman-Hart Alexion Derralynn Hughes Cystic Fibrosis Trust

Spina bifida, a group of rare neural tube defects, affects many thousands of infants globally. #HarringtonScholar surgeon-scientist Diana Farmer UC Davis Research UC Davis Surgery is developing new therapeutic approaches using stem cells 👇👇👇

M. Peter Marinkovich, MD, David Hilliard, a leading researcher and #HarringtonScholar, discusses the promising potential of pz-cel for treating severe RDEB. #RareDisease #GeneTherapy bit.ly/3WleFt8

A new paper from Carlo Rinaldi Carlo Rinaldi, who leads the OHC neurological disorders theme 👇The team shows how innovation using microRNA can address cell specificity and off-target effects, which are current hurdles in gene editing technologies for the treatment of #rarediseases

A limited number of spaces remain - join us for our in-person symposium on drug development for #rarediseases presented by leaders in academia, industry and patient representation. Summary - Oxford-Harrington Rare Disease Centre  Symposium 2024 (cvent.com)

It's two weeks until the OHC Symposium! Session 2 RARE THERAPIES LAUNCH PAD features Dan O'Connor ABPI, Tim Yu Harvard Medical School, Richard Scott Genomics England, and Dame June Raine MHRAgovuk. The panel discussion will include Kath Bainbridge Department of Health and Social Care and Mel Dixon Cure DHDDS