Now we are all settled in after Christmas and the New Year, here at the AKU Society we would like to take the opportunity to look at all our plans for 2019. From trips aboard to new research, it seems like 2019 will be as busy as ever.At the end of the month, the AKU Society team and trustees will travel to the historic Italian city of Siena. There, we will attend the last project board meeting of the
DevelopAKUre consortium and the 11th scientific workshop on AKU. The past meetings have been a way for researchers involved in the DevelopAKure trials to get together, talk about their work and expand the scientific understanding of AKU.
The AKU Society will be there to highlight the ways we have supported patients enrolled in the trials and the wider community, while planning how we will support patients after the trial. One of the major areas that will be discussed is how the AKU Society will help get nitisinone licensed as a treatment for AKU and how we can help patients access it.
The end of January also marks the end of the patient element of the Sonia 2 clinical trial. Once completed, the data will be analysed. If positive, SOBI (Swedish Orphan Biovitrum), who make the drug, will begin the process of applying for a license for the drug to be made available to all AKU patients in Europe. We expect this to be completed in 2023.
With the end of one research project, we are at the start of another. This year will see the beginning of the SOFIA Paediatric study, designed to better understand at what age the symptoms of AKU start in children under 16. Once this is known, we will have a better understanding of when children should start the drug, so they grow up with no symptoms of the disease at all.
Our 7th patient workshop will take place in Autumn this year. The workshop will focus on an often-requested topic, diet. The date and location of this workshop have yet to be decided. Watch this space for more information.
In February the team will be attending the various
Rare Disease Day receptions at the capitals of all the home nations. We are sure to meet some fascinating people and learn about the government’s plans for rare diseases in 2019. We will update everyone about what we discovered via a blog.
We still have spaces available to run the Cambridge half marathon in March. The very popular run sold out in days when tickets were released. It’s easy to see why it’s so popular: a mostly flat route through the beautiful and historic streets of Cambridge. What better way to raise money for AKU?
Head here for more information.We will also be hosting out 4th Stand up for AKU comedy night. This year the night will take place in Liverpool, the AKU Society’s historic home. More information on date and location will be released as soon as we begin to plan the night.
To end the year, we will be holding a truly international Garrod Day celebration. We will be encouraging patients and friends of the AKU Society across the world to hold coffee mornings and other fundraising events to mark Sir Archibald Garrod’s birthday on 25th November. For more information please email juliet@akusociety.org.
The above events are only a fraction of the plans for 2019. Make sure you follow us on Twitter and Facebook and subscribe to our E-newsletter to hear the latest from the AKU Society.