We believe the ACT model with physician led care is the best course of action in treating patients. We strongly encourage everyone to take a look at SB882

Hello, my name is Kasey Feldt and I reside in Franklin, Virginia and my son was diagnosed with Krabbe Leukodystrophy. Krabbe Disease is currently not on the Virginia Newborn Screening Panel is currently in RUSP consideration. It is imperative for Krabbe Disease to be added to the VA NBS for early detection and a life-altering transplant. My son, Dawson Luke Feldt, was a fighter. He started showing symptoms at 3 months of age when he never gained the milestone of head control. Over the months, he had feeding difficulties and lost his ability to smile and laugh. After an MRI with anesthesia, he started having spasms. At 6 ½ months old, we received the devasting news of the diagnosis of Krabbe Disease. Hearing your son will not live past the age of 2 years old is a feeling that cannot be described. We were told to grieve the life we dreamed of having and to make memories, take pictures, and love him his final days. My son suffered with secretions, nerve pain, breathing difficulties, spasms, loss of mobility, and on November 8, 2020 he lost his battle at 15 months old. I have fought multiple times to get Krabbe Disease added to the Virginia Newborn Screening panel but since Virginia is not RUSP aligned it is not approved. I am happy to report that Krabbe Disease was added to the RUSP in 2024. The next step is for Virginia is to be RUSP aligned so that Krabbe Disease and other disorders can be added to Virginia’s Newborn Screening Panel. I am asking you to support House Bill 1782 for Virginia to be RUSP aligned. With Krabbe Disease Newborn Screening, a transplant happens within the first month of life and it extends the child’s life expectancy. There is no cure yet, but what I would not give to have more time with my son. I am asking lawmakers to support HB 1782 in requiring the state to automatically consider adding disorders/diseases on the RUSP to the Virginia Newborn Screening Panel. With rare diseases, time is of the essence and early detection is key. I hope my son’s story has touched your hearts. He was a fighter until the end, and I promised him I would always fight and advocate for those with rare diseases. Thank you, Kasey Feldt

Subject: Passage of HB 1782 We are Jim and Vicki Danoy. We live in Fairfax County and are proud residents of the Commonwealth of Virginia. We are also grandparents to Sofia, a beautiful little girl born in the Commonwealth on 14 September 2021. At the age of six-months Sofia was diagnosed with Krabbe Disease, a form of Leukodystrophy, and given two-years to live. As you may know, Leukodystrophy is a heritable degenerative neurological disease involving the “white matter” covering nerve cells. While there is currently no cure, early detection—that is at birth, is key to mitigating the worst effects of the disease and at least providing a better quality of life for those afflicted by this terrible disease. This requires newborn screening for Krabbe Disease and other forms of Leukodystrophy. Last year, Krabbe was added to the Recommended Uniform Screening Panel (RUSP) by the U.S. Department of Health and Human Services (HHS), a major breakthrough brought about by intense lobbying efforts, by many groups and individuals including our daughter and her husband. We urge the Virginia General Assembly to pass HB 1782 which would: • Require the state to automatically consider screening newborns for any disorder on the RUSP, removing the burden on patient communities to initiate the process. • Require the Department of Health to evaluate a disorder’s inclusion within 12 months and implement testing within 36 months if deemed appropriate. • Ensure resources are available to facilitate the addition of new disorders by codifying the Department’s existing authority to charge a reasonable fee for the tests. Making testing for Krabbe Disease a standard part of newborn screening in the Commonwealth is essential to detection and mitigation. Sofia has defied the odds and has lived to celebrate her 40th month of life. However, sadly Sofia’s life will be short because at the time of her birth the Commonwealth did not require screening for Krabbe Disease and therefore any hope of mitigating the degenerative effects of the disease had passed. But as Sofia’s grandparents we believe by implementing uniform newborn screening we can help future parents and grandparents from enduring what we have endured and give Sofia’s life meaning. Sofia’s parents are Virginia residents and proudly serve our nation—her mom, our daughter, works in the Intelligence Community and her dad, our son-in-law, is a Commander in the Navy. They are hard-working individuals with a deep religious faith. They have never asked for anything that they themselves have not been willing to give. We ask that you as legislators and Governor Youngkin do everything in your collective power to bring about uniform testing for Krabbe Disease and other forms of Leukodystrophy to the Commonwealth with the passage of HB 1782. Thank you for your attention in this important, life-giving matter. Sincerely, Jim & Vicki Danoy Herndon, VA

Dear Committee Members, My name is Elsa Kendall, and I live in Arlington, Virginia. I am a college freshman, an amateur cartographer, and a future archivist. Most importantly, I am an advocate. When I began showing troubling bruises shortly after birth, my parents became concerned that I had inherited hemophilia, a rare genetic bleeding disorder, from my father. However, despite continuous bruising and bleeding episodes, I was not tested by doctors. It was not until six years later, after a particularly nasty fall that resulted in extreme bruising, that I received a hemophilia diagnosis. In the six years between my initial symptoms and my diagnosis, I had a multitude of injuries that could’ve been treated with hemophilia medication but were not due to a lack of diagnosis. If there had been an established protocol for me to be tested as a newborn, my diagnosis would easily have been discovered; achieving diagnosis earlier would have significantly lessened the impact of prolonged bleeding on my health. I urge you to support HB 1782 to align Virginia’s newborn screening standards with the Recommended Uniform Screening Panel (RUSP). My story is an extremely fortunate one. Though I sustained injuries and experienced episodes that I would not have with a diagnosis at birth, the effects of delayed diagnosis on my body have been much less severe than what those with other rare, heritable diseases and disorders may experience. RUSP alignment guarantees that families will be better prepared and better equipped to combat the effects of rare diseases. It also means that families will not be forced to experience the anxiety, fear, and financial consequences that accompany delayed diagnosis. In passing HB 1782, please join me in advocating for everyone affected by rare disease: parents, caregivers, siblings, grandparents, and patients. We are all connected by similar stories; however, this bill has the power to change our collective narrative. Thank you for your time. Sincerely, Elsa Kendall

Comments Document

Dear Chair Sickles, Vice Chair Tran and Honorable Committee Members, On behalf of the Immune Deficiency Foundation (IDF), and the community affected by primary immunodeficiency (PI) in the Commonwealth of Virginia, I write to you to provide comments and express support to HB1782. This legislation would require Virginia to screen newborn children for any disorder on the federal Recommended Uniform Screening Panel (or RUSP). IDF is dedicated to improving the diagnosis, treatment, and quality of life of people impacted by PI by fostering a community empowered by advocacy, education, and research. Individuals with PI have one of the over 450 rare disorders in which a person’s immune system fails to function properly because of genetic or intrinsic defects. They are highly susceptible to recurrent, persistent, and severe infections, which, without treatment, can lead to organ damage and often require significant interventions and hospitalization. Fortunately, most people with PI can live healthy, productive lives if they receive lifelong immunoglobulin replacement therapy, an innovative and lifesaving therapy derived from donated plasma. Severe combined immunodeficiency (SCID) is one of the most severe types of PI. A child born with SCID completely lacks a functional immune system and is extremely vulnerable to severe and life-threating infections. Without treatment, these children typically do not survive past two years of age. Critical to early treatment is the newborn screening blood test that supports diagnosis. If a child with SCID is diagnosed and treated within the first few months of life, their long-term survival rate is more than 90%. Thanks to newborn screening for SCID in all fifty states, most children with SCID are diagnosed before they develop health problems. SCID is just one of the conditions on the RUSP and innovative technology will allow for screening of more diseases in the coming years. We urge you to support HB 1782 to ensure that as new diagnoses are added to the RUSP, Virginia will have the resources and planning in place to ensure that babies born in the commonwealth will be screened for these conditions. Matthew Prentice, Director of State Policy with the Immune Deficiency Foundation.