We are all desperately waiting for a treatment for Dutchtype CAA (or "the Plug thing") and hoping for a future without strokes for ourselves, our family members, our children and grandchildren. We think that we have reason to be hopeful.
In 2021 we started TRACK DCAA. A natural history study to learn more about the trajectory of the disease in gene carriers and ways to best measure this. That information is vital for a potential drug trial, because 2 things are central to a trial: 1) When do we need to start the intervention? and 2) How do we measure if the drug does what we hope it does - slown down disease progression?
TRACK DCAA is sponsored by two major pharmacompanies: Alnylam and Regeneron. For good reason: they are developing the drug ALN-APP. ALN-APP is an RNA therapy that potentially slows down the production of toxic amyloid by masking part of our DNA. The companies are very interested in developing the drug for everyone with a form of CAA.
The patient advocates of the DCAA association are working together with research teams in the Netherlands and Perth to convince Alnylam to include DCAA family members in a trial with ALN-APP. This means that gene carriers have early access to a drug that potentially slows down Dutchtype CAA and is a huge opportunity for everyone who is a gene carrier or might be one. It also means that it gets more likely that there is a treatment in our future, which is huge for current AND future generations.
Our patient advocates (Sanne and Dini) together with Perth's leadership team (Ralph, Kevin, Hamid and Samantha) would like to invite you to our webinar. We will update you about TRACK DCAA, inform you about ALN-APP and tell you more about how you can help make a trial happen.
The meeting will be online. We will share a link to the meeting ASAP. During the meeting there will be plenty of time to ask your questions. If you have any questions beforehand that you want answered during the webinar, please inform us through Messenger or email Sanne (email@example.com). See you soon!