These are definitely exciting and hopeful times for Rett community. Taysha Gene Therapies have announced that the first patient was dosed with the investigational gene therapy, TSHA-102, in the REVEAL Adult Study, a clinical trial for females 18 years and older living with Rett syndrome. As things move forward, we´d like to share the letter […]
We are delighted that the Rett Syndrome Europe Network Support and Research Event was so successful. Many thanks to our funders The European Joint Programme for Rare Diseases Network Support Scheme and our sponsors Anavex, Taysha, Neurogene and Neuren for making this possible. Also thanks to our country hosts in Turkuye, Hungary, UK, Netherlands, Serbia, […]
Katharina is now 14 ½ years old and has been epileptic since the age of three. In the beginning, the epileptic seizures manifested themselves at long intervals and were somewhat under control with medication. However, as she grew up, the seizures became more frequent and intense. Katharina also developed therapy resistance to all the medications […]
Since Neurogene received a lot of questions from the Rett community about the upcoming gene therapy trial they announced earlier this year, they prepared a letter with some answers to the frequently asked questions. Please read the document bellow.
Clinical-stage biopharmaceutical development company Neurotech International (ASX: NTI) has launched a new clinical program into the use of its daily oral cannabinoid-based drug to potentially treat Rett Syndrome in female patients. The program will comprise a filing for Human Research Ethics Committee (HREC) approval and the commencement of a phase two clinical trial investigating the […]
I came into the world on an afternoon in May 2018. May, the 1st to be exact. No Labor Day for my parents that year. After a rough arrival, I extended my stay at the hospital due to an infection. The first of a long series… My name is Matteo Covini, I am 4 years […]
RSE sent a joint letter with Rett UK to Rett Syndrome News to address concerns that their articles do not recogise the fact that some males with Rett syndrome can and do survive beyond infancy. Thank you to Kate Cahill Ferdinandsen for bringing this matter to our attention and providing links to relevant research articles. […]
We are delighted that the Rett Syndrome Europe Network Support and Research Event was so successful. Many thanks to our funders The European Joint Programme for Rare Diseases Network Support Scheme and our sponsors Anavex, Taysha, Neurogene and Neuren for making this possible. Also thanks to our country hosts in Turkuye, Hungary, UK, Netherlands, Serbia, […]
Spring 2022 has been very productive season for Rett scientists bringing hope to our families. We are happy to share some exciting news with our community. 29th March 2022 Taysha gene therapies annoucned that their Clinical Trial Application (CTA) had been approved by Health Canada. TSHA-102, which utilises the novel miRARE platform to regulate transgene expression […]
Inna is 9 years old and lives in France with her family. Her mother, Elsa Delhelle Gasnier, says that since Inna was diagnosed with Rett syndrome, their family schedule got filled with never ending medical appointments, which many times feel like a stressfull battlefield: “Appointments, appointments, always appointments… Good news and bad news… When we […]
