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
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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 a physician-scientist and geneticist whose
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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 him into Salt Lake City
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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 muscle tone. From the beginning,
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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
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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 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 By Stella Ekaputri Click Here to View Full Article
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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 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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