Emily Whitehead was the first-ever pediatric patient to receive a CAR-T cell therapy after rounds of chemotherapy failed to treat her leukemia.
On Tuesday, we will hear from Emily and her father, Tom, about their quest to ensure other patients can benefit from CGTs. #ATMPEurope
There are already some #GeneTherapies approved for #RareDiseases in the EU, such as:
🧬Skysona - Children with early cerebral adrenoleukodystrophy
🧬Libmeldy - Forms of metachromatic leukodystrophy (MLD)
🧬Zolgensma - Spinal muscular atrophy
#RareDiseaseDay2022@CGTCatapult
What are cell and #GeneTherapies?
➡️The delivery of a gene to modify a defective gene directly to the patient through an injection
➡️Extracting & adapting cells, protein or #DNA from a patient or donor to provide personalised therapies with potential to address #RareDiseases
What do we do?
Our team of experts collaborate with academia, industry & health care providers to develop new, life-changing cell and #GeneTherapies
Learn more 👉 https://t.co/WkupFqVYCW
💬 Matthew Durdy, CEO, @CGTCatapult#RareDiseaseDay#RareDiseaseDay2022
Hello, Matthew Durdy here, CEO of @CGTCatapult 👋
CGTC is a non-profit, independent innovation & technology organisation helping cell & #GeneTherapies be safer, more effective & affordable.
Ask any questions you have about these therapies👇
#RareDiseaseDay#RareDiseaseDay2022
'Absolute revolution': UK biotech firms thrive despite Brexit threat. @CGTCatapult was featured in @guardian over the Christmas break - https://t.co/RCZDRAnykr
Hear what @Jeremy_Hunt, Secretary of State for Health and Social Care, has to say about govt plans for adolescent #mentalhealth, in the final episode of #StormandStress on @BBCRadio4
Listen live at 11am as @drsallymarlow puts the questions to Mr Hunt: https://t.co/Zv2hOwT90W
Take a listen back to the latest episode of Storm and Stress on @BBCRadio4 - a programme that explores the ways we look at #mentalhealth among young peope:
https://t.co/SAi2uQ8ZQE