Victoria Arteaga MBA, is the Directora para America Latina Fondo Syngap, co-founder of SHER Sociedad Hispana Enfermedades Raras, and mom to 7-year old Syngapian Amelia. Nancy Kessler is an educational specialist, community psychologist and caregiver/sibling to her 66-year old Syngapian sister, Caren. Both are Syngap Research Fund board members.
SRF attended the Advanced Therapies Week conference in Miami, Jan, 18-20, 2023. Both Vicky Arteaga and Nancy Kessler were excited to represent SRF. While attending workshops on patient and clinical considerations, Vicky and Nancy met with key innovators and decision makers in biotech and rare diseases.
This conference was an incredible experience, and the technological advancements we learned about are happening right now! This is an exciting time for SYNGAP1 and the SynGAP Research Fund, as science is accelerating for other diseases such as SMA, Parkinson’s Disease and Angelman. SRF is ahead of the curve as a small rare disease group, and it was apparent at this event. Only 5% of rare diseases currently have therapies, and with SRF’s natural history studies, we are making great progress in the requirements for clinical trials. To move the clinical trial process forward, there is a new FDA program called ARC, Accelerating Rare Cures. Enhanced tools are available, and patient reported outcomes are vital during FDA review. The FDA process can move along faster for cell and gene therapy approval if we have a great natural history study, which we are on the road for right now! We are confident that SRF patients are on track while participating in Ciitizen, Rare-X and Simons Searchlightdatabases!
In addition to uncovering where we stand in the journey to a cure, conferences like these are truly enlightening. We were thrilled to have attended and met with others sharing common interests while learning about these groundbreaking medical advancements. We encourage any and all rare disease family members, researchers and scientists to attend these amazing learning experiences. Even if the science is complex and dense, don’t despair. If you’re in a room full of scientists, you’re in the right place. It’s so important to make connections with special people in this industry. You can gain so much more from a conference than just scientific knowledge!
The future is bright and cures are on the horizon!
Here is a list of conferences you may want to attend with SRF this year:
- NIH Rare Disease Day / Feb 28
- RDW with Everylife in DC
- Drug Dev with GG/ Philly / May 1-3
- Segundo Congreso Scientífico Syngap1 online May 13, 2023
- National Congress of Pediatric Neurology in Mexico – Chihuahua/ May 15-20
- WODC / Gaylord National Resort & Convention Center Washington, D.C. / May 23-25
- DC / NINDS Nonprofit Forum July 2023
- ILAE / Ireland / Sept 2-6 /
- DEI / Global Genes 09/18/23
- San Diego / Global Genes / 09/19/2023 – 09/20/2023
- Strategy, Advocacy & Partnering for the World Orphan Drug Industry, Barcelona / 30 Oct – 2 Nov
- SRF SYNGAP1 Conference 2023 Florida / SRF in November 30 – December 1
- AES / Florida / December 1- 3