In 2025, we are proud to continue our long-standing support of Dr. Angela Lin and her team at Massachusetts General Hospital, and to announce the launch of two newly funded clinical sites: one at Stanford Medicine Children’s Health and the other at Texas Children’s Hospital.

To provide the FDA with a comprehensive understanding of Myhre syndrome, including the lived experiences of patients and caregivers, the community met with the FDA in December 2024. The full report is out now.

We are excited to announce the launch of our new partnership with Emory University designed to shed light on the underlying molecular mechanisms of Myhre Syndrome and lay the foundation for future treatments.

Genetically modified T cell therapies have been in the news for their ability to improve outcomes for cancer patients, and a recent longitudinal study confirmed that serious complications were extremely rare.

Congratulations to Dr. Callewaert and Pr. Cormier-Daire, who have won pretigous awards for their rare disease research.

We are proud to announce a $450,000 grant to fund a groundbreaking research project designed by Genethon, collaborating with Professor Valérie Cormier-Daire (Genomic Medicine Service for Rare Diseases, Necker-Enfants Malades Hospital) in France.

We’re thrilled to announce a special Rare Disease Day 2025 event—the Myhre Masterpieces Art Auction! We’re inviting members of the Myhre community to create one-of-a-kind artworks that will be featured in our art auction.

We’re thrilled to announce the opening of a dedicated Myhre Syndrome Clinic at Stanford University’s Cardiovascular Connective Tissue Clinic in California, USA. This clinic brings together experts in genetics, cardiology, and other specialties to provide personalized care for patients and families while advancing research into Myhre syndrome.

Last year, we were approached to submit an essay for the American Journal of Medical Genetics discussing the work we’re doing at the foundation. Kate Wears, our Executive Director, jumped at the chance to raise awareness and share the foundation’s mission with a wider audience.