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Message from Jill Kelly
On February 9, 2023 the Advisory Committee on Heritable Disorders in Newborns & Children (ACHDNC,) a federal committee that makes recommendations to the Secretary of Health about newborn screening (NBS), voted whether to add Krabbe Disease to the Recommended Uniform Screening Panel (RUSP). Despite the irrefutable evidence, there was a tied vote. Therefore, Krabbe won’t be added to the RUSP. This decision will keep most states from adding Krabbe to their NBS panels. This also means that children with Krabbe will continue to suffer and die from a treatable disease when born in these states.
For almost two decades, the Hunter’s Hope Foundation has been fighting for NBS for Krabbe Disease. Krabbe is a truly horrible and deadly disease. If babies are not screened for the disease at birth through their state’s newborn screening program, they will be diagnosed too late for life saving treatment and typically die by the age of two. In their short lives, Krabbe robs these children of the ability to speak, walk, swallow, and virtually all voluntary movement while causing extreme complications and painful suffering.
The Secretary of Health makes the final decision on what diseases are included on the RUSP. Secretary Becerra has the power to change this.
Children with Krabbe Disease deserve to live.
You can help! Take action here.
Help us convince Secretary Becerra to right this terrible wrong and to do his part to make sure all U.S. children are screened for Krabbe Disease at birth.
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)
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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