Top 5 ASF Accomplishments of 2023
To close out the year, we want to briefly reflect on some of the milestones that mark our progress in 2023. These advancements were made possible by the engagement and support of the ASF Community.
- Launch of the ASF Alport Patient Registry
In August, we launched our patient registry, which is now fully funded and administered by ASF. Based on guidance from experts in Alport research, we believe this is the most critical way U.S. patients and caregivers can advance research that may lead to potential treatments and/or a cure.
- Expansion of the Alport syndrome pharmaceutical pipeline
This year, ASF established relationships with a wide range of industry partners from across the world working toward novel Alport treatments and/or a cure – these include preclinical studies, to those preparing to launch in 2024! Our investment in collection of human data (via the patient registry and NEPTUNE natural history study) are important tools we’ve established and invested in to support the growing pharmaceutical industry pipeline.
- Held Alport Connect 2023 in person in San Diego
In October, we held our first in-person meeting since the start of the pandemic. This was also our largest in-person event to date – hosting patients, family members, and experts in the field from around the country. We will share initial details about our 2024 meeting early next year.
- Growth in membership
This year, we welcomed 556 new members to our community – all of whom are invested in awareness, understanding, and advancement of research in Alport syndrome. Additionally, our international, moderated Facebook support group for patients and family members grew by over 630 users since January 1st.
- Awards for young Alport patients
During the 2023 Paul Silver Enrichment Award cycle, we were fortunate to receive applications from a tremendous group of young adults living with Alport syndrome. Due to their outstanding applications, seven individuals were awarded funds provided by the Silver Family, ASF, and several anonymous donors. We are honored to have the opportunity to support their interests and individual goals.
ASF Board of Directors Welcomes New Members
As we look ahead to 2024, the ASF Board of Directors is pleased to announce the addition of two new members to help lead its efforts.
Daniel Kilpatrick is a Partner at the investment firm Crestview Partners, where he heads its investments in financial services companies. He lives in New York City with his wife Sarah and two children. Multiple members of Daniel’s family have Alport syndrome, and he is proud to support ASF in its mission to improve the lives of all those affected by Alport syndrome.
December West has served on the Akron Children’s Hospital Parent Advisory Council, Ohio Children with Medical Handicaps Parent Advisory Council, Ohio Rare Disease Advisory Council, and Akron Children’s Health Collaborative as a board member. She lives with her husband and five children in Akron, Ohio. December has been a fierce advocate for her daughter, Nhalani, who has autosomal recessive Alport syndrome. Representing ASF at several important meetings with industry and researchers, both have contributed their perspectives on life with Alport syndrome as a patient or caregiver.
We appreciate Daniel and December taking on new leadership roles for ASF.
Patient Registry Enrollment Update
As of December 1, 2023, data for 319 Alport syndrome patients has been captured through participation in the ASF Alport Patient Registry.
Additionally, dozens of enrollees have uploaded genetic testing results files (in the form of JPEGs, PNGs, and PDFs) to the platform, allowing for the documentation and future study of various genetic mutations.
The ASF Alport Patient Registry is private, free of charge, completely voluntary, and open to individuals living with Alport syndrome in the U.S. (and its territories and outlying islands) at all ages and stages of disease (pre- and post-transplant). Parents/caregivers can also enroll their child/children with Alport syndrome in the registry.
Thank you to all those who have chosen to participate and update their data over time!
New Animal Model for Alport Research
While mouse, zebrafish, and canine models have all been used in scientific research to understand more about our disease, there may be unique utility in studying the disease in an amphibian model.
As axolotls are already a staple of biological research labs around the world, a recent article in the Journal of Biological Chemistry explains how axolotls can serve as novel models of Alport-impaired kidneys to better our scientific understanding of the disease.
MAC Member Spotlight: Dr. Carter
Dr. Caitlin Carter is double certified in adult and pediatric nephrology and is one of the newest members of ASF’s Medical Advisory Committee (MAC). She has already been involved in crafting our patient resources and participating in many sessions at Alport Connect 2023.
We asked Dr. Carter what drew her to the Alport community, her interest in research, and why she is hopeful for the future of Alport care.
Click here to read the full Q&A with Dr. Carter.
View our Newsletters and Announcements page for an archive of prior communications.
