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

News, Research /

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

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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 /

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

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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

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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 /

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)

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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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New Global Patient Registry for MicrogliopathiesGlia

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GliaConnect has launched a new global patient registry for individuals and families affected by microgliopathies such as ALSP, BANDDOS, and Nasu-Hakola Disease. In partnership with Sanford CoRDS, this registry helps bring together patients, families, and researchers to advance understanding and treatment of these rare disorders. The data collected will help identify symptoms, improve care management,

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Positive Results Announced for Alexander Disease Clinical Trial

Clinical Trials, News /

Ionis anounces positive topline results from pivotal study of zilganersen in Alexander disease The Ionis Zilganersen Team shared a message with the Alexander disease community: September 22, 2025 Dear Alexander disease community, Ionis announced today positive initial results from the pivotal clinical trial (NCT: 04849741)1 of zilganersen (ION373) in people with Alexander disease (AxD). In

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