This morning’s #59events 🚲: a breathtaking sunrise. Event 27. 31x 59km to go, the last event being a 322km 🚲. Raising 💵 for MPS Society UK, here for those with rare diseases and #YoungLivesvsCancer. #charity #cycling #mps #4thechildren #fabrydisease #kindnessmakesadifference #thanksforyoursupport 🙏

I’m not really surprised at these findings: Fabry disease is still underdiagnosed, especially among women, a new study reports. fabrydiseasenews.com/news/fabry-dis…

2023 Orphan Drugs: PDUFA Dates and FDA Approvals Click on the link below👇 to view the list of important regulatory dates for all orphan drugs for 2023. checkrare.com/2023-orphan-dr… #rarediseases #drugapprovals

ST-920, a potential gene therapy for Fabry disease, has been granted fast track designation by the U.S. Food and Drug Administration. fabrydiseasenews.com/news/potential…

Blood levels of periostin, a protein associated with kidney injury, may be a valuable marker for kidney damage in Fabry disease, a study says. fabrydiseasenews.com/news/blood-per…

Looking for abnormal, clumped features called mulberry bodies in a person's urine may help in the diagnosis of Fabry disease, a study found. fabrydiseasenews.com/news/mulberry-…

Do you have a rare disease or care for someone who has a rare disease? Here’s an important survey about #newbornscreening from EURORDIS-Rare Diseases Europe and Genetic Alliance UK: tiny.cc/survey_NBS_RD. Make your voice heard. #raredisease #Fabrydisease #genetics #genomics

Don’t miss these #GenomicsConversation sessions with NHS East Genomics team @Kate_Sci and our Chair Gemma Chandratillake exploring #RareDisease on Wed & #Genomic medicine for your patients on Fri

It's time for a #GenomicsConversation. Thank you Genomics Education for all the work you are doing to raise awareness and understanding of #genomics and #healthcare.

There are 42 integrated care systems across England, covering populations of around 500,000 to 3 million people. But why are they needed, and how do they work? Read our ICS explainer to find out. kingsfund.org.uk/publications/i…

#RAREsummit23 is an incredible opportunity to bring the #raredisease community together & the Newcastle Mito Team are excited to be involved because we are definitely #StrongerTogether 💪🏻💚 NIHR Newcastle BRC NCL_RareDisease JWMDRC The Lily Foundation Newcastle Mitochondria and muscle pathology Muscular Dystrophy UK

Calling all healthcare professions – why not begin your genomics knowledge journey today with a short course? This Genomics 101 module introduces the testing process, including why, when and how genomic testing is done: orlo.uk/OZ1Nt

'The New Opportunity to Help Rare Disease Patients'🧡 The article from MedCity News discusses investment in rare diseases and how global spending on rare diseases is estimated to reach $260 billion by the end of 2025. Read more here: ow.ly/sKCV50Plsoe

Lots happening in #rarediseaseresearch. #raredisease #research ukri.org/news/new-14-mi…

It’s great news that there’s another treatment for #Fabry. However in what context is ‘a small co-pay’ applicable on the #NHS? Elfabrio was approved in the U.K. fabrydiseasenews.com/news/elfabrio-…

A HUGE thank you to all our amazing media partners helping us get the word out about #RAREsummit23 - We’ll see you tomorrow! bit.ly/RAREsummit23

Thursday 12 October is RARESummit - the bi-annual rare disease event organised by Cambridge Rare Disease Network (CamRARE) . Really looking forward to hearing from policy-makers, researchers, patient voices and innovators. If you can’t be there you can follow #RAREsummit23

Promising gene therapy trial results for #FabryDisease: One-year data indicate ST-920 stabilized kidney function, reduced disease severity, and improved life quality. fabrydiseasenews.com/news/interim-t… #genetherapy #Fabry #raredisease

We've been working with patients, families & clinicians to develop a health & care #RareDisease passport template to help communicate key medical & care information. The phase 2 report is just out! Cambridge Rare Disease Network (CamRARE) #RareDiseaseDay2024 #RareDiseaseDay #RDD2024 #RDD

How time flies! Fabry Awareness Month 2024 is coming to an end: t.ly/K6pDh #Fabry #Raredisease #FabryAwareness