Year: 2025

Finding Answers, Finding Community: Tilyn’s Journey with TUBB4A Leukodystrophy  

Give 2025, News, Share Your Story / December 17, 2025

Finding Answers, Finding Community: Tilyn’s Journey with TUBB4A Leukodystrophy My name is Miesha Thomas, and I’m the mom of Tilyn Sincere, my 6-year-old son living with TUBB4A Leukodystrophy (H-ABC). Our journey started with years of questions and a misdiagnosis of cerebral palsy. I knew something wasn’t right, he fell often, army-crawled sideways, and missed milestones …

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Finding Our Village: Our Journey with Alexander Disease

Give 2025, News, Share Your Story / December 17, 2025

Finding Our Village: Our Journey with Alexander Disease My name is Deb, and together with my husband, Jim, we are the proud parents of Daniel. From the very beginning, I sensed something was not right during my pregnancy. Despite being reassured repeatedly that everything was fine, I trusted my instincts and sought out a neonatologist. …

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Research Summaries December 2025

Clinical Trials, Research / December 19, 2025

ALEX TLC (Alex, The Leukodystrophy Charity) produces monthly research summaries highlighting recent leukodystrophy research and clinical trials, with a focus on key findings and advancements in treatment. These summaries are prepared by volunteer Biomedical Science students, who provide their interpretations of the latest studies and findings — helping make complex research more accessible to the …

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ULF Awards $33,000 Research Grant to Advance Gene Therapy for H-ABC

News, Research / December 19, 2025

The United Leukodystrophy Foundation (ULF) is proud to announce the award of a $33,000 research grant to Dr. Jun Xie, PhD, of UMass Chan Medical School, to support an innovative preclinical gene therapy project focused on Hypomyelination with Atrophy of the Basal Ganglia and Cerebellum (H-ABC). About the Funded Project Project Title:AAV-mediated gene silencing for …

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Historic Expansion of Newborn Screening Includes MLD

News / December 18, 2025

Historic Expansion of Newborn Screening Includes Metachromatic Leukodystrophy (MLD) We are encouraged to share that the U.S. Department of Health and Human Services (HHS) has officially added Metachromatic Leukodystrophy (MLD) — along with Duchenne Muscular Dystrophy (DMD) — to the federal Recommended Uniform Screening Panel (RUSP) for newborn screening. This decision was announced by HHS …

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Eden’s Story: Helping Families and Supporting Research Through Her Legacy

Give 2025, News, Share Your Story / December 17, 2025

Eden’s Story: Helping Families and Supporting Research Through Her Legacy https://ulf.org/wp-content/uploads/2025/12/Eden.mp4 We are the Murphy Family, and our daughter, Eden Alexandra, was born on January 10, 2025. She was not breathing at birth, and that was the start of her short but complicated journey. She was in the NICU for thirty-three days and fought through …

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Hope in Action: ULF Board Member’s Son Receives World’s First SLC6A1 Treatment

News / December 11, 2025

We are excited to share a powerful new People Magazine feature highlighting our ULF Board Member Amber Freed and her son Maxwell, and the extraordinary journey that led to a groundbreaking milestone in rare disease history. In the article, Amber’s son Maxwell is described as having a neurodevelopmental condition known as SLC6A1, a rare genetic …

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Strength for Today, Hope for Tomorrow: Laila’s Story

News, Share Your Story / December 8, 2025

Strength for Today, Hope for Tomorrow: Laila’s Story https://ulf.org/wp-content/uploads/2025/12/Laila-2.mp4 We are Sam and Angela, and this is the story of our daughter, Laila. Our journey with leukodystrophy began when our daughter, Laila, was diagnosed with MLC1 with subcortical cysts at just 2 years old. Today, she is 14. She began walking independently, though with a …

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A Family’s Journey with ALD – Robert & Nancy

Give 2025, News, Share Your Story / November 17, 2025

Robert & Nancy: A Family’s journey with ALD When a family faces a leukodystrophy diagnosis, the journey can feel overwhelming and isolating. That’s where the United Leukodystrophy Foundation (ULF) comes in. Since 1982, ULF has provided families with life-changing information and resources, access to expert care, and a compassionate, connected community that ensures no one …

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New Global Guidelines Issued for Vanishing White Matter (VWM)

News / November 19, 2025

New Global Guidelines Issued for Vanishing White Matter (VWM) — A Significant Step Forward for Families and Care Teams We’re pleased to share a major advancement in the care of VWM. A new international consensus document has just been published, offering evidence- and expert-based recommendations for the diagnosis and clinical management of VWM. This is …

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