Resources for patients
We know it can be frustrating waiting on new treatments
We are working hard for rare disease patients.
Is the MRC CoRE providing therapies?
No. We are working to understand how these therapies might be delivered, and exploring the strengths and weaknesses of various tools, but we are not (yet) ready to deliver these therapies to humans.
We are not ready to work with specific patient cases yet – but if you would like to get involved we highly recommend you get in contact with one of the patient advocacy groups below.
We will work with those groups when we are ready to start engaging with patients.
What resources exist for patients and their families?
UK NHS knowledge hub for rare disease patient advocacy groups: https://www.genomicseducation.hee.nhs.uk/genotes/knowledge-hub/rare-disease-patient-advocacy-groups/
Umbrella groups
- Syndromes Without A Name: https://undiagnosed.org.uk/
- UNIQUE (rare chromosomal disorders support group): https://rarechromo.org/
- Rare as One Network (supporting patient-led organizations working to improve the lives of people affected by rare disease): https://chanzuckerberg.com/science/programs-resources/rare-as-one/
- Genetic Alliance Patient Empowerment Group: https://geneticalliance.org.uk/campaigns-and-research/making-our-communitys-voice-heard/patient-empowerment-group/
You may also be interested in the Rare Rebels storytelling project: https://rarerebels.com/
- UPNAT (UK Platform of Nucleic Acid Therapy for rare disease treatment): https://rd-research.org.uk/node/upnat/
- N=1 Collaborative (working to make individualised medicines possible): https://www.n1collaborative.org/
- Oxford Harrington Rare Disease Centre (seeking to bring 40 drugs into clinical trials by 2035): https://www.oxfordharrington.org/
- Dutch Center for RNA Therapeutics (working to provide RNA therapies for disorders of brain, spinal cord and eye): https://www.rnatherapy.nl/contact