Ionis Astro-AxD Study
ASTRO-AXD STUDY IIONIS is inviting people living with Alexander Disease and who are starting or are already being treated with Zanvastro (Zilganersen) and their caregivers to participate in a voluntary observational study. Please review the Astro-AxD Study information for more details Click Here for Full Press Release
2026 ULF Moser Service Award: Aurora Pujol, MD, PhD
At the United Leukodystrophy Foundation’s 2026 Scientific Symposium & Family Conference, ULF was honored to recognize Aurora Pujol, MD, PhD, FACMG with the 2026 Moser Service Award, presented in grateful appreciation for her outstanding service and commitment to the lives of people with leukodystrophy and their families. Dr. Pujol is…
Paxton’s Story: My Angel, My Heart, My Everything
Paxton’s Story: My Angel, My Heart, My Everything My name is Seanna, and my whole world is my grandson, Paxton. He is 3 years old, has AxD, and it has devastated us. We just thought Paxton was speech delayed. Then one day, he started falling every 10 minutes. We rushed…
Manaka’s Journey: Finding Hope in the Unknown
Manaka’s Journey: Finding Hope in the Unknown My name is Megumi, and my daughter Manaka was born in April 2018 by cesarean section because she was breech. There were no complications during pregnancy, and no abnormalities were noted when she was born. However, Manaka hardly cried and had weak overall…
Researchers at the ALC discover a new leukodystrophy
Researchers at the ALC discover a new leukodystrophy From Amsterdam Leukodystrophy Center Newsletter September 2026 Researchers at the ALC, working with international partners, have discovered a new leukodystrophy. So far, the disease has been described in three patients. Click here for full article
JLR Research Article: Spatial and temporal brain biodistribution of neuropathogenic sphingolipids of Krabbe disease
JLR Research Article: Spatial and temporal brain biodistribution of neuropathogenic sphingolipids of Krabbe disease Authors: Tingting Yan, Shih-Chang Hsueh , Salma Begum , Narayana Chelakkara Venkiteswaran, Justin Ellenburg, Boone M. Prentice, and Gustavo H. B. Maegawa Click Here for Full Article
iScience Article: Sphingolipid-neutralizing molecular thereapy reduces psychosine cytotoxicity in Krabbe Disease
iScience Article: Sphingolipid-neutralizing molecular thereapy reduces psychosine cytotoxicity in Krabbe Disease Authors: Salma Begum, Shin-Change Hsueh, Eara M.Y.Cheria, ….,Michael H. Gelb, Change-Chun Ling, Gustavo H.B. Maegawa Click Here for Full Article
Journal News article: Mapping toxic lipid buildup in a rare neurological disease
JOURNAL NEWS article: Mapping toxic lipid buildup in a rare neurological disease By Stella Ekaputri Click Here to View Full Article
Finding Answers, Finding Community: Tilyn’s Journey with TUBB4A Leukodystrophy
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,…
Finding Our Village: Our Journey with Alexander Disease
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…
ULF Awards $33,000 Research Grant to Advance Gene Therapy for H-ABC
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…
Historic Expansion of Newborn Screening Includes MLD
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…
Eden’s Story: Helping Families and Supporting Research Through Her Legacy
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…
Hope in Action: ULF Board Member’s Son Receives World’s First SLC6A1 Treatment
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…
Strength for Today, Hope for Tomorrow: Laila’s Story
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…
A Family’s Journey with ALD – Robert & Nancy
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…
New Global Guidelines Issued for Vanishing White Matter (VWM)
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…
New Global Patient Registry for MicrogliopathiesGlia
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…
In Memorial and Honor: May – September 2025
ULF received donations in memorial or honorarium for the following during May through September 2025. We apologize for any inadvertent omittance or misspellings. Please notify us if we have made a mistake at office@ulf.org Honoring Loved Ones Through Generosity We are deeply grateful for the generous donations made in honor…
Teagan’s Story
A Mother’s Love, A Child’s Legacy: Teagan’s Story Our daughter, Teagan Ann, will forever be the strongest person I have ever known. She was born on November 13th, 2016. A perfectly healthy, beautiful baby girl. Ten fingers and ten toes. Our little miracle. Time went by so quickly, and Teagan…
Positive Results Announced for Alexander Disease Clinical Trial
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…
Kim’s Story
Kim’s Story: A Family Bond with ALSP In 2019, my brother Jeffrey Cade was 42 when he awoke one morning unable to speak clearly. Jeffrey immediately called our family, and we urged him to drive to the hospital. After an overnight stay, the doctors assumed Jeffrey had a minor stroke and…
Moa’s Story
Moa’s Story Moa is a fantastic girl with a big, bright smile that charms every soul she meets. A girl with her heart in the right place and a twinkle in her eyes. She leaves no one untouched. We are from Sweden and live in Malmö. Moa is nine years…
The Hidden Impact of Vanishing White Matter (VWM) on Families
New Research Sheds Light on the Burden for Parents, Siblings, and Partners A groundbreaking international study has revealed something our community has long known: Vanishing White Matter (VWM) doesn’t just affect patients. It deeply impacts their families too. A new study, “The impact of vanishing white matter on unaffected family…