Hi Cure,

Did you miss the webinar with Dr. Elizabeth Berry-Kravis, one of the principal investigators for the Roche Program? Don’t worry, we saved the recording and you can watch it below.



If you have updates you would like to be included in next week’s newsletter, please email us at info@cureangelman.org.

We are beyond excited to announce the 2023 CAN Grand Prize trip location:

The top 8 CAN fundraisers and 2 random winners who meet the eligibility criteria will receive an all-expenses-paid trip to Dr. Scott Dindot’s lab at Texas A&M in College Station, TX and see incredible science in real time.

Dr. Dindot and his lab created the first large animal models of Angelman syndrome, using the pig. In addition, they are the pioneers who studied the UBE3A gene for many years to understand its evolution, as described in his recent paper in Science Translational Medicine, to reach human clinical trials for Angelman syndrome! 

This is a once-in-a-lifetime opportunity to tour this cutting-edge lab and get a view behind-the-scenes of all of the incredible work being done in the Dindot Lab. 

The CAN Campaign closes October 31, 2023 at 11:59pm EST. It’s not too late to get started on your fundraising efforts. If you need any tips to get started, email fundraising@cureangelman.org! 

Create a fundraising page today:

Thank you to those who attended the joint FAST | ASF webinar with Dr. Elizabeth Berry-Kravis, one of the principal investigators for the Roche (Tangelo) program. We are grateful for Dr. Berry-Kravis to share her thoughts and experience regarding clinical trials for Angelman syndrome and other rare diseases. 

We are currently triaging the final questions received to Roche. We hope that by facilitating these opportunities for you to share your concerns and questions has provided some clarity and understanding on how clinical trials work for rare diseases.

FAST and ASF are committed to moving forward from this setback as we both continue to fight for a better future for our loved ones living with Angelman syndrome. 

For those who missed it, here is the link to the recording:

Ultragenyx sent a letter to both FAST and the ASF to share with the community in response to the recent Roche program update. 

You can read the letter from Ultragenyx here:

Welcome, Keith Sutton PhD, to the FAST Scientific Advisory Board!

Keith is the director of pre-clinical development at Resolution Therapeutics. He is a translational scientist with decades of experience developing novel cell, gene, and immunomodulatory therapies for rare disease and is a true expert on the process of advancing a promising candidate for a therapy to a first-in-human drug. Dr. Sutton is a huge asset to the Angelman community as we push toward the clinic for so many more shots-on-goal.

Learn more about our Scientific Advisory Board:

Project WellCAST is back and recruiting rare disorder caregivers!

Eligible participants will receive FREE THERAPY OR RESOURCES, available 100% via telehealth, as part of this research study!

Project WellCAST is a NIH-funded clinical trial investigating which telehealth-based therapies and resources best meet the needs of rare disorder caregivers like you.

Learn more:

Please note: Because this is a research study, qualifying participants do not select which type of support they will receive. Participants may stop the study at any time without penalty. This is a research study funded by the National Institutes of Health and approved by the Purdue University Institutional Review Board.

Thanks for reading, and please share this link with anyone in your network who would be interested in receiving our weekly newsletter.