AGS Community Exceeds Expectations: Pausing the Biogen Patient Registry Study

Update: Our patient registry project with Biogen has been completed as of January 2024 and we are now pivoting to our AGSAA Contact Registry and a partnership with Ciitizen to create an AGS Natural History Study by using patient medical records to unlock key insights and data for research and treatment opportunities - both launching April/May 2024.

Dear AGS Community,

We are absolutely thrilled to share some electrifying news with all of you today – the Biogen Patient Registry Study has exceeded our wildest expectations! Our community has rallied together, and we've already collected so many responses!

This remarkable response is a resounding statement. It's proof that our AGS community is far larger and more organized than many previously believed. It's a dynamic testament to our shared commitment to pushing the boundaries of AGS research and advocating for the betterment of all affected by this condition.

As we pause the study to analyze this wealth of data, we want to extend our heartfelt gratitude to each and every one of you who participated. Your unwavering support and active involvement have set a new benchmark for what's possible in AGS research. Together, we're shaping a brighter future for our community. With your energy and dedication, the sky's the limit for what we can achieve together!

Introducing the Faculty Members of the Aicardi-Goutieres Syndrome ECHO Program

Exciting News! We are thrilled to unveil the exceptional team of experts who will be leading the way in our Aicardi-Goutieres Syndrome ECHO (Extension for Community Healthcare Outcomes) program. Together, they bring a wealth of knowledge and experience to this groundbreaking initiative, dedicated to improving the lives of individuals affected by AGS and advancing healthcare.

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Meet Our Esteemed Faculty:

Min Ae Lee-Kirsch, MD

Dr. Lee-Kirsch, a clinical geneticist, leads a research group at the Technische Universität Dresden that specializes in investigating the molecular mechanisms behind autoinflammation and autoimmunity. Her research particularly focuses on perturbations of the type I interferon (IFN) axis, which includes conditions like AGS. Additionally, Dr. Lee-Kirsch provides clinical care and treatment to AGS patients, combining her expertise in research and patient care for a comprehensive approach to the disease.

Stephen Wong, MD

Dr. Wong, a pediatric rheumatologist at Seattle Children's Hospital, specializes in treating inflammatory brain disorders like AGS. He brings extensive expertise to the care of multiple AGS patients at Seattle Children's Inflammatory Brain Disorders Clinic, providing essential support and treatment for those in need. Dr. Wong's dedication and qualifications make him a trusted resource for this specialized field of pediatric medicine.

Laura Adang, MD, PhD, MSTR

Dr, Adang, an attending physician in the Division of Neurology at Children's Hospital of Philadelphia (CHOP), specializes in the comprehensive care of children diagnosed with leukodystrophies. Her regular leadership in managing AGS patients underscores her commitment to providing the highest level of medical care in this field. Dr. Adang's expertise is a valuable resource for AGS patients and their families seeking expert guidance and treatment.

Davide Tonduti, MD, PhD

Dr. Tonduti, the founder and medical coordinator of COALA (Center for Diagnosis and Treatment of Leukodystrophies and Genetic Leukoencephalopathies), is an expert in the field of leukodystrophy treatment. Notably, he also serves as a principal investigator in an AGS clinical trial, demonstrating his commitment to advancing research and improving the lives of individuals affected by this condition. His multifaceted role ensures comprehensive care and cutting-edge treatment options for patients.

Nicole Jaffe, MD

As a complex care pediatrician at the Children’s Hospital of Philadelphia’s (CHOP) Leukodystrophy Center, Dr. Jaffe treats many children with AGS. Her approach is rooted in providing comprehensive and holistic care, prioritizing each child's ability to thrive and excel, irrespective of their condition. Dr. Jaffe's dedication ensures that AGS patients receive the support they need to lead fulfilling lives.

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Our faculty members are committed to empowering healthcare professionals to enhance the care and quality of life for those affected by AGS. Together, we can make a difference!

Stay tuned for updates, schedules, and valuable resources coming your way in 2024. We're excited to embark on this journey of knowledge sharing and collaboration together. 💪

Contribute to AGS Clinical Trial Development: Join the Biogen Study

Update: Our patient registry project with Biogen has been completed as of January 2024 and we are now pivoting to our AGSAA Contact Registry and a partnership with Ciitizen to create an AGS Natural History Study by using patient medical records to unlock key insights and data for research and treatment opportunities - both launching April/May 2024.

Update: in light of an astonishing response, we’ve paused the Biogen patient registry study. See our update.

AGS Families,

We have an opportunity for you to make a meaningful impact on the development of an AGS clinical trial. We'd like to invite you to participate in a patient registry study sponsored by Biogen aimed at understanding AGS better and improving the clinical trial development process.

💡 Why Participate?

Make a Difference: Your valuable insights will contribute to advancing a Biogen clinical trial in AGS and potentially lead to a more effective and convenient clinical trial.

$50 Amazon Gift Card: As a token of appreciation for your time, Biogen is offering a $50 Amazon gift card to everyone who completes the survey.

30 Minutes Well Spent: The survey will only take about 30 minutes of your time, but the impact it can have on AGS research is immeasurable!

👉 How to Participate:

1. Click on the survey link below to get started:

2. Complete the survey with your honest responses.

3. Share this information with other AGS families to maximize our collective impact.

🌐 Learn More:

To delve deeper into the world of AGS research and clinical trial development, consider these resources:

• AGS Patient Registry: Stay connected with the AGS community and access valuable information. Link to AGS Patient Registry.

• Biogen Webinar: Discover more about Biogen's commitment to AGS research in our first webinar. Link to the Webinar.

Let's unite as a community and contribute to a brighter future for those with AGS. Your involvement is significant, and together, we can make a real difference.

Thank you for being the incredible AGS advocates that you are. Let's spread the word and make AGS history together!