The MED13L Foundation Nominated for the 2026 Prix Galien USA Best Patient First Award in Honor of Michael J. Fox Recognition honors the Foundation’s patient-led research ecosystem, “From Families to Frontiers,” which has built an integrated drug-discovery pipeline for MED13L syndrome. CHICAGO, IL, September 14, 2026 — The MED13L Foundation has been nominated for the…
The MED13L Foundation has completed a five-year analysis of MED13L registry data from Simons Searchlight, led by Ricardo Ramirez, the Foundation’s Chief Scientific Officer. Drawing on 17 quarterly reports from 2021–2026, this analysis provides our clearest view yet of how the MED13L community has grown and what we are learning about the shared clinical features…
In June 2026, the MED13L Foundation surveyed families about their children’s use of NSAIDs, including ibuprofen, following repeated reports of changes that appeared to extend beyond expected effects on fever or pain. While this small, family-reported survey cannot establish efficacy or causality, the responses provide an important signal that warrants systematic investigation. 17 families responded,…
Collaborative initiative will evaluate an innovative RNA-based strategy to restore gene expression and accelerate therapeutic development for MED13L Syndrome and other rare neurodevelopmental disorders. CHICAGO, IL – August 5, 2026 – The MED13L Foundation today announced its participation in a collaborative research initiative led by COMBINEDBrain and Johns Hopkins University (JHU) to evaluate a novel RNA-based…
A message from Ricardo Ramírez, PhD, Chief Scientific Officer of The MED13L Foundation — with the latest updates on our progress and achievements. Now available! Get the latest update in your language and share it with your community below.
Friday, December 5th, 2025 The MED13L Foundation is pleased to announce the execution of a Sponsored Research Agreement with the University of California, San Francisco (UCSF) to support a new study titled “CRISPRa Proof of Concept Constructs for MED13L Haploinsufficiency.” This agreement provides $165,000 in support for a two-year research period. The project will explore…
As featured in Brain & Life Magazine BOSTON, May 5, 2025 — The MED13L Foundation today announced a nearly $250,000 grant to help launch an innovative natural history study at Boston Children’s Hospital’s Rosamund Stone Zander Translational Neuroscience Center (RSZ TNC). The MED13L Syndrome Investigation of Natural History and Development (MIND Study) is a three-year…
One step closer to a therapeutic treatment. Could it be this easy? We Have a Hit! Rarebase Delivers. It’s not a home run, but a solid double from Rarebase! After a long couple of years and two rounds of testing, naproxen was shown to upregulate MED13L mRNA in MED13L patient-derived neurons
The Finding a Drug Discovery Partner (First) Edition of the Newsletter (10/17/21) Hello CURE MED13L Community! I’m Nick Seaver and this is the inaugural edition of the CURE MED13L newsletter. I can’t tell you how thrilled I am to be kicking this off. Since the MED13L Family and Research Virtual Meeting in August, we have made tremendous strides pushing…
A week ago we hit a high point for the MED13L Foundation. The wonderful research team at Combined Brain completed their many month review of all things MED13L Syndrome and presented their strategic plan. It gives a super helpful roadmap for the things we want to do in order to set ourselves up for clinical…
Denver, Colorado, December 4th, 2026. This December, MED13L families will gather in person at the Grand Hyatt Denver. The day brings our community together around the latest MED13L research and the chance to spend real time with one another. Early bird pricing available!
