Many thanks to Niki and Petros who live in Cyprus for sharing their wonderful story.
Good news from Neurogene as they progress their gene therapy trial to the next stages. You can read their letter to the community here.
You can read the full article below:
Many thanks to Future Rare Diseases for sharing this publication with us. You can read the full paper here.
To celebrate Rare Disease Day 2024 one of our board members Bojana Milanov was interviewed by NORBS – the National Organisation for Rare Diseases of Serbia. Here we share her story. Many thanks Bojana. A rare disease means any disease that occurs in less than 5 people per 10,000 citizens, but a number of rare […]
Becky Jenner, President, had the pleasure of meeting Julia Ramlogan a couple of years ago when Julia launched her new organisation to raise awareness and help progress towards a cure. We are delighted to share Julia’s story and progress with her mission. https://www.montana-zug.ch/en/news/january-2024/parent-feature-interview-with-a-super-mom-julia-ramlogan
Dr Tim Benke, Medical Director of the Rett Center of Excellence at Colorado Children’s Hospital is the lead Principal Investigator in a study that is seeking to understand the lived experience of Males with Rett syndrome via their parent report. He would love to have responses from parents in Europe COMIRB: 23-0075PI: Dr. Tim BenkeV. […]
Official press release from Taysha giving more detail of their REVEAL paediatric gene therapy trial with sites in the US and UK.
Taysha announce details of their REVEAL paediatric study in the US which is now recruiting patients.