Today, January 30, 2024, in a landmark moment, the Federal Newborn Screening Advisory Committee voted to add Krabbe Disease to their Recommended Newborn Screening Panel. This decision, a result of over twenty years of unrelenting advocacy, brings us a step closer to our collective aim of screening every newborn for Krabbe Disease.
This victory is a testament to the tireless efforts of the Hunter’s Hope Foundation, the Kelly Family, the Krabbe Community, and a beacon of hope for children who are affected by this disease in the future.
With newborns now being screened for Krabbe at birth, this opens the door to early detection and lifesaving treatments.
We are genuinely moved by the support we’ve garnered in achieving this milestone and continue to steadfastly work towards our mission for all Leukodystrophies.
We invite you to join us in our mission to improve the lives of these courageous children battling for their future.
You can support our Newborn Screening advocacy efforts here.
You can learn more about the Federal Newborn Screening Advisory Committee here.
Supporting Information
Hunter’s Hope Nomination Cover Letter for Krabbe Disease to be added to the HRSA ACHDNC RUSP
RUSP Nomination Packet for Krabbe Disease HRSA Revisions 090321 (Data as of September 2021)
Hunter’s Hope Response to the ACHDNC “No” Decision – Letter to Secretary of Health (March 3, 2023)
Hunter’s Hope Response to the ACHDNC Reasons for “No” Decision – Letter to ACHDNC Committee Chair (April 19, 2023)
Hunter’s Hope Second Letter to ACHDNC Chair (May 30, 2023)
Hunter’s Hope Revised Nomination Letter to the ACHDNC (July 22, 2023)
Click here to Take Action
History
When the federal Advisory Committee on Heritable Disorders for Newborns and Children (ACHDNC) was formed in 2006, they created the Recommended Uniform Screening Panel, also known as the RUSP
The RUSP was created to be a minimum standard of diseases that should be included in a state’s newborn screening panel and are purely a recommendation, as each state maintains autonomy as to how many diseases it includes in its newborn screening program. However, states are often unwilling to add diseases that are not on the RUSP, therefore, this process is crucial to the advancement of Krabbe newborn screening
Since the RUSP was created in 2006, the ACHDNC has only recommended the addition of eight diseases to the RUSP. Ultimately the U.S. Secretary of Health must approve the committee’s recommendations, and, to date, has not veered from the committee’s advisement on this matter.
Krabbe NBS and the ACHDNC
In 2007, Hunter’s Hope nominated Krabbe Disease for inclusion on the RUSP, and it was not recommended by the committee at a vote of 8 to 7. The committee identified 3 evidence gaps that needed to be filled. In the years that followed, Hunter’s Hope has teamed up with medical and scientific experts to ensure the gaps identified by the committee were filled and that these advancements were published in peer reviewed medical and scientific journals
After years of persistent effort, Hunter’s Hope, together with a team of medical and scientific experts, submitted the nomination package for Krabbe Disease to be included on the RUSP in June 2021. In May 2022, the ACHDNC voted unanimously to advance Krabbe Disease to full evidence review. Shockingly, on February 9, 2023, the committee’s vote on Krabbe Disease resulted in a tied vote. There were many procedural and factual errors throughout the meeting and we are fighting this decision. Learn how you can help us in this fight here.
The history of this process can be found here.
Below are some of the families who have helped us by providing public comments to the committee.
Public Comments
Click on the link below to read some of the Public Comments that have been made by families affected by Krabbe Disease. If you read them, you will understand the urgency of taking action to ensure every child is screened at birth for Krabbe Disease.
Click here to read Public Comments from Families