Rare Trait Hope Fund

04/16/2026

AGU gene therapy clinical trial is starting soon:

04/08/2026

Investigational New Drug application has been approved for AGU gene therapy by the FDA. Clinical trial and patients' enrollment will start soon in the USA.

03/22/2026

Monthly parents' meeting will be help tomorrow, Sunday March 22. Please, check the correct time for the meeting. Some places changed clock to the summer time, others not yet. Time of the meeting is 11 am New York time. The link is posted in the AGU parent Slack group. Let me know if you cant access the invite.

02/22/2026

Monthly parents' meeting will be help tomorrow, Sunday February 22. Usual time and place. Let me know if you need an invite.

10/26/2025

Just a quick reminder: the monthly AGU meeting will be held on Sunday, October 26. It will start at 11 am New York time. Please convert it to your respective times. See you there!

08/21/2025

Almost a month has passed and this Sunday (August 24, 2025) is the global AGU meeting. The zoom invitations is the same as the last one. Do you have it? If not, please reach out.

This Sunday we will discuss the upcoming gene therapy clinical trial in detail (a few doctors might be present on the call to answer questions).

Translations will be available with the help of AI.

07/23/2025

This Sunday (July 27, 2025) is the global AGU meeting. Zoom invitations were sent to over 60 people. Did you get one? If not, please reach out. Translations will be available with the help of AI.

06/21/2025

Many of you are joining the meeting tomorrow and got an invitation. Please, send email to info (at) raretrait.com if you want to join and don't have an invite yet. The Zoom meeting it tomorrow (Sunday) at 11 am US Standard time or 17:00 European Central time.

02/28/2025

This is the message for our Scandinavian AGU community. The meeting will be held this Sunday, 6 p.m. Helsinki time with translations to Finnish and Swedish. We will discuss AGU gene therapy, clinical trials, Natural History study, etc. Please, email [email protected] for a link to this Zoom meeting if you would like to participate.

Another meeting will be held in 2 weeks for the global AGU. More updates will be posted shortly.

02/04/2025

Great meeting with the Finnish AGU parents group. I was overwhelmed by all the help, support, patience and interest in the meeting. We will make these meetings regular. Thank you for everyone that participated!

12/10/2024

Rare Trait Hope Fund, in partnership with National Organization for Rare Disorders, Inc. (NORD)®, has launched the Aspartylglucosaminuria (AGU) Registry and Natural History Study. This registry gives patients worldwide a platform to share their experiences and information about AGU, a rare disease that causes developmental, speech, and motor delays. Currently, there is no cure for AGU. Designed with input from scientists and patients, this global resource aims to provide data for researchers to advance drug development and improve patient care.
“The launch of the AGU Registry is a significant milestone in our fight against this ultra-rare disease. By sharing their experiences, participants will help accelerate research efforts to develop much-needed treatments and improve the quality of life for those with AGU. The success of combating AGU for every current or future patient depends upon the community's participation in this registry,” said Julia Taravella, Executive Director of Rare Trait Hope Fund.
Learn more and join the AGU Registry at https://AGU.iamrare.org.

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12/03/2024

We are thrilled to share the release of "Nazira: Purrpose like no other" book, a heartfelt journey narrated by Nazira—witty and wise Persian cat who has been by the side of Daniel and Alexander from diagnosis till now. This unique meow-moir offers an intimate glimpse into a family journey trying to create a life-changing treatment for their sons and others with an ultra-rare genetic condition, Aspartylglucosaminuria (AGU). This book is shares not only their struggles but also love and humor that you cant live without in these situations.
Every dollar from the book (available globally) go directly to Rare Trait Hope Fund to pay for the drug and to finance clinical trials for AGU. With your support, we’re closer to offering a cure—for children around the world with this devastating condition. Our first goal is to offer treatment to 10 children. By purchasing, sharing, or donating (this Giving Tuesday too), you’re helping us take a giant leap toward saving children’s lives.
➡️ Get your copy of the book today:

Meet Nazira, a brilliant and empathic cat who shares her unique perspective on life with two brothers battling an ultra-rare genetic disease, Aspartylglucosaminuria (AGU). In this heartfelt memoir, Nazira narrates the inspiring journey of their parents’ relentless quest to secure a diagnosis and ....