A Joyful Laugh, a First Step, a New Future

This end-of-year giving season, we are reaching out with a powerful message:

Progress is measured in moments of hope: a family finding support, a new research study launching, or a grant that accelerates our path to treatments. Because of your generosity, the FAM177A1 Research Fund has turned these milestones into real, measurable progress for families impacted by FAM177A1-Related Neurodevelopmental Disorder (FRND).

For moms like Janet, whose daughters Bella and Emma live with FRND, progress looks like simple joys that once felt out of reach. Her daughter, Emma, recently learned to walk at age 7 and said her very first word. “Her voice comes through in different ways,” Janet shared. “You have to listen with patience, but when she laughs, it fills the room!” 

A mother's heart recognizes joy in these hard-won moments—and that joy is the hope that your support creates.

At the FAM177A1 Research Fund, we work tirelessly toward treatments for Emma and Bella, for our children Charlotte and Cooper, and for every family still waiting for answers. We are racing against the clock; the hard-won developmental progress of our children is at risk of slipping away. Every moment counts.

That’s why we’re funding bold research that brings us closer to treatments:

• Yale University: Dr. Berrak Ugur and her team are using patient cells to uncover how the gene FAM177A1 shapes brain development.

• University of Portsmouth, UK: Dr. Annie Godwin and Rosie Currams are studying Xenopus (frogs!) to unlock the mysteries of FAM177A1 and to test potential treatments.

• Hebrew University: Professor Rami I. Aqeilan, PhD is pioneering an AAV9-based gene therapy for (FRND), aiming to deliver a functional copy of the essential FAM177A1 gene and transform the lives of affected children and families.

  
  

While we fund groundbreaking research, we also stand shoulder-to-shoulder with families living with this ultra-rare disease by connecting them to resources, care, and community. As Janet says, “My hope for Emma is that the world sees her just like I do: understood, loved, supported, and full of potential.”

Your year-end donation makes this possible. Your support drives discoveries for Emma and Bella, for Charlotte and Cooper, and for all our FRNDs who deserve a future full of laughter, progress, and joy. 

Please support FAM177A1 with a donation. Thank you for fueling progress and for being part of this unstoppable community of hope. You remind us that joy is part of the cure.

With love and deep gratitude,

Jill Hawkins

Founder & President

FAM177A1 Research Fund