Dear NUCDF Community,

There is a lot to share this month, including an important update about RAVICTI availability that we want to make sure reaches every family. We also have exciting milestones from the Partner Network, new ways to lend your voice to UCD research, and everything coming in October.

NUCDF has been informed that Amgen plans to retire branded RAVICTI® (glycerol phenylbutyrate) oral liquid on December 31, 2026. This is an important change for our community, and we encourage families currently using branded RAVICTI to begin conversations now with their metabolic team, specialty pharmacy, and insurance provider.

NUCDF is closely monitoring this transition and will continue to share updates as more information becomes available.

Please read our full statement for important details and next steps:
nucdf.org/generic-medications

Already using a generic? Share your experience through the NUCDF Generic Medication Survey.

NUCDF has partnered with Children's Hospital of Philadelphia (CHOP) on a five-year, $38.9 million federal grant from ARPA-H to develop personalized CRISPR gene editing therapies for severe urea cycle disorders. The project is led by Dr. Rebecca Ahrens-Nicklas at CHOP and Dr. Kiran Musunuru at the University of Pennsylvania. It is part of ARPA-H's broader $160 million THRIVE program.

As a subaward partner, NUCDF will bring the community's voice into the research through engagement, education, and stakeholder input. This is a meaningful step forward for the UCD community, and we are proud to be part of this work.

Read the full announcement: NUCDF Joins ARPA-H Initiative

One year ago, we launched the NUCDF Partner Network, a PCORI-funded initiative to bring together patients, caregivers, clinicians, researchers, dietitians, and industry partners to build the first patient centered research roadmap for urea cycle disorders.

The results from Year One exceeded every goal we set:

Year Two is now underway. We are analyzing the community's input from focus groups, drafting a research needs list, and preparing a prioritization survey so that your voice directly shapes where UCD research goes next. The final roadmap will be shared with researchers, funding agencies, and industry partners.

Read the full Year One report: Year One of the NUCDF Partner Network

The FDA is planning a Patient Listening Session focused on children under 12 with urea cycle disorder (UCD), and we are looking for parents and caregivers who can share what managing this condition requires.

This private, virtual session will bring together a small group of caregivers and FDA staff. You would have about 5 to 10 minutes to describe your family's experience: the demands of daily care, the risks, difficult decisions, and what improvements would matter most. No medical training or public speaking experience is needed. You know your child best, and we will help you prepare.

Your firsthand perspective can help FDA staff understand what clinical data alone cannot: what children with a UCD and their families need, and what meaningful improvements would look like in everyday life.

If you are interested, or would like to learn more, contact Greg Trevors at gjtrevors@gmail.com.

Marsha Michie and Megan Allyse are recruiting participants for the CONNECT Study, an NIH-funded partnership between NUCDF and Case Western Reserve University. The study is designed to hear directly from people in the urea cycle disorders community about their perspectives on emerging therapies, including gene editing, and how these treatments should be approached now and in the future.

Your experiences and opinions matter. By participating, you can help researchers better understand what patients and families value, what questions and concerns they have, and what should be considered as new therapies are developed.

Interested? Sign up here: CONNECT Study Survey

NUCDF contributed to two research posters presented at the 2026 Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium in Helsinki, Finland.

A new poster explored headache as a potentially overlooked early symptom of urea cycle disorders. Among the findings: 80% of registry respondents with late onset UCDs reported headache as an early symptom, and 57% of OTC carriers reported recurrent headaches. The research grew out of a social media conversation between Dr. James Nurse, Dr. Radha Ramadevi, and NUCDF staff, with Dr. Andrea Gropman confirming the clinical findings.

Learn more and read the poster

A validation study of the MyRareDiet™ app, led by Melanie Gillingham, PhD, RD, of Oregon Health and Science University, found that protein tracking in the app agreed strongly with the traditional dietitian administered 24 hour recall method. The study included 31 participants with UCDs, organic acidemias, and MSUD. This work was co-authored by NUCDF and supported by NIH and OCTRI grants. 

We dedicate this work to the memory of dietitian Debra Geary Hook and former NUCDF executive director Cynthia Le Mons, whose vision made MyRareDiet possible.

Learn more and read the poster 

Check Ammonia Awareness Month

October is Check Ammonia Awareness Month, and our community plays an essential role in making the message heard.

Throughout the month, we will share family stories, clinical perspectives, research, and resources that reflect the real impact of delayed recognition and the importance of acting quickly.

We are asking families, clinicians, researchers, and partners to help us expand the reach of the campaign. Visit CheckAmmonia.com to access the awareness toolkit, download social media resources, share campaign content, and add your own voice using #CheckAmmonia.

You can also request printed Check Ammonia resources to share in your local community, clinics, schools, hospitals, or other places where greater awareness can make a difference.

Every share helps put this message in front of someone new.

Help us make Check Ammonia impossible to miss this October.

Request materials: Check Ammonia Materials Form
Toolkit: Checkammonia-toolkit | CheckAmmonia.com

Cure the Cycle Challenge

October also brings the 12th Annual Cure the Cycle Challenge, NUCDF’s annual community fundraising campaign.

This year’s event takes place October 10, 2026, in Napa, California, with opportunities to participate virtually from anywhere. Whether you ride, walk, run, create a team, fundraise, or simply help spread the word, everyone can be part of Cure the Cycle.

Funds raised through this year’s challenge will benefit the Cindy Le Mons Fellowship Grant, helping support and develop the physicians and researchers who will advance UCD care and research for the next generation.

Our goal is $100,000, and every participant, team, donation, and shared story brings us closer.

Together, we can Cure the Cycle.

Register, create a team, or start your fundraising page at charity.pledgeit.org/curethecycle.

The Brave Little One Foundation, created by the family of Pruitt Kruse, is hosting a coloring contest for ages 3 to 5, 6 to 8, and 9 to 12. Each winner chooses a nonprofit to receive a $100 donation in their honor. Mail entries to PO Box 51, Lennox, SD 57039 or email a photo to bravelittleonefoundation@gmail.com. Include first and last name, phone number, and age. Downloadable coloring sheet can be found here: Download File

Thank you for being part of this community. Every voice, every story, and every mile makes a difference. We are grateful to have you with us.