ALEXANDER DISEASE CAREGIVER Information
Just Worldwide is conducting a market research study on behalf of a pharmaceutical company. The study aims to gather insights from caregivers of people with Alexander Disease to better understand their experiences and opinions. The research involves a 40-minute online survey. Participants who complete the survey will receive $100 as…
In Memorial and Honor: February 2025
ULF received donations in memorial or honorarium for the following during the month of February 2025. We apologize for any inadvertent omittance or misspellings. Please notify us if we have made a mistake at [email protected] or by calling 815-748-3211 In honor of Daniel Nathan Horton In Loving Memory of Daniel….
FDA Approves Ctexli (Chenodiol) for Treatment of Cerebrotendinous Xanthomatosis (CTX)
Mirum’s CTEXLI™ (chenodiol) Tablets Receives FDA Approval for Treatment of Cerebrotendinous Xanthomatosis (CTX) Mirum Pharmaceuticals has announced that the U.S. Food and Drug Administration (FDA) has approved Ctexli (chenodiol) tablets as the first treatment for cerebrotendinous xanthomatosis (CTX), a rare and progressive genetic disorder that affects cholesterol metabolism. CTX leads…
The ULF Board of Directors’ statement regarding the changes to healthcare and research because of reduced NIH funding.
The United Leukodystrophy Foundation Board of Directors’ statement regarding the changes to healthcare and research because of reduced NIH funding. February 24, 2025 “The United Leukodystrophy Foundation (ULF) is deeply concerned about proposed changes in healthcare and research. These changes may limit patient access to life-saving treatments; may impact the…
STUDY: Rescue of in vitro models of CSF1R-related adult-onset leukodystrophy by iluzanebart: mechanisms and therapeutic implications of TREM2 agonism
Understanding CSF1R-Related Leukodystrophy and a Potential New Treatment: Iluzanebart This study titled Rescue of in vitro models of CSF1R-related adult-onset leukodystrophy by iluzanebart: mechanisms and therapeutic implications of TREM2 agonism and published by Journal of Neuroinflammation, explores CSF1R-related adult-onset leukodystrophy with axonal spheroids and pigmented glia (CSF1R-ALSP), a rare neurodegenerative…
FDA Approves First Treatment for CTX, a Rare Lipid Storage Disease
PRESS RELEASE: FDA Approves First Treatment for Cerebrotendinous Xanthomatosis, a Rare Lipid Storage Disease The U.S. Food and Drug Administration (FDA) has approved Ctexli (chenodiol) as the first treatment for cerebrotendinous xanthomatosis (CTX), a rare lipid storage disorder. CTX is a genetic condition caused by mutations in the CYP27A1 gene,…
STUDY: The natural history of variable subtypes in pediatric-onset TUBB4A-related leukodystrophy
Understanding TUBB4A-Related Leukodystrophy in Children: A Study on Disease Progression and Symptoms This study titled The natural history of variable subtypes in pediatric-onset TUBB4A-related leukodystrophy and published in Molecular Genetics and Metabolism, focuses on TUBB4A-related leukodystrophy, a rare genetic disorder that affects the brain’s white matter (the part of the…
Study: Impaired Peroxisomal Beta-Oxidation in Microglia Triggers Oxidative Stress and Impacts Neurons and Oligodendrocytes
Research Summary: Impaired Peroxisomal Beta-Oxidation in Microglia Triggers Oxidative Stress and Impacts Neurons and Oligodendrocytes In this study, titled Impaired Peroxisomal Beta-Oxidation in Microglia Triggers Oxidative Stress and Impacts Neurons and Oligodendrocytes, and publshed by Frontiers in Molecular Neuroscience, investigates how dysfunction in peroxisomes — small cell structures that break…
28th ASGCT Annual Meeting- Empowering Patients 2025: A Cell and Gene Therapies Summit
Hosted by American Society of Gene + Cell Therapy This inaugural virtual event March 12 and 13, 2025 (9 a.m. – 12:30 p.m. ET on both days), will provide timely education and engaging discussions on cell and gene therapies (CGTs). In alignment with ASGCT’s strategic plan, the goal is to empower advocates,…
In Memorial and Honor: January 2025
ULF received donations in memorial or honorarium for the following during the month of January 2025. We apologize for any inadvertent omittance or misspellings. Please notify us if we have made a mistake at [email protected] or by calling 815-748-3211 In honor of Bob Hodge Jr., Brian Greene, Garner Hicks, and…
In Memorial and Honor: October – December 2024
ULF received donations in memorial or honorarium for the following during the months of October through December 2024. We apologize for any inadvertent omittance or misspellings. Please notify us if we have made a mistake at [email protected] or by calling 815-748-3211 In honor of Adam Sledzik Stanislaw Sledzik CTX is…
A Legacy of Hope: Join Bob & Yvonne in Supporting the ULF
We are Bob and Yvonne Rauner. We are the parents of our two sons, Kevin and Paul, who had adrenoleukodystrophy and adrenomyeloneuropathy. Our story began with all the same challenges ALD families faced at the time. We navigated a three-year diagnostic odyssey with Kevin. He was “diagnosed” with ADHD and…
Finding Strength in Community: Jen’s Story
I’m Jen Martin-Cutler, proud mom to Clark, my handsome ginger son diagnosed with Pelizaeus-Merzbacher Disease (PMD) in December 2015 at 11 months old. Clark’s leukodystrophy journey began with challenges like delayed milestones, difficulty swallowing, and nystagmus, leading us to specialists at Lurie Children’s Hospital. His care team included experts in genetics,…
Living with ALD: A Journey of Strength and Community
My name is Margaret Halter. My husband, John, was diagnosed with Adrenoleukodystrophy (ALD)/Adrenomyeloneuropathy (AMN) in 1989 at the age of 31. We’ve been married for 41 years, live on a beautiful farm in Ohio, and have two children, a daughter and a son, both with families of their own. Our daughter…
Minoxry Therapeutics: Leriglitazone has met the primary endpoint in NEXUS, the pivotal trial for pediatric patients with cALD
Press Release Summary (December 11, 2024): Minoryx Therapeutics, a late-stage biotech company, and Neuraxpharm Group, a European specialty pharmaceutical firm, announced positive outcomes from the NEXUS trial, a pivotal 96-week study evaluating leriglitazone in pediatric patients with cerebral Adrenoleukodystrophy (cALD). The trial demonstrated a significant 35% arrest of disease progression…
A Special Year-End Message from Dr. Mousumi Bose
My name is Mousumi Bose, and I am the mother of Ilan Betzer. Ilan was born in August 2010 and was soon diagnosed with Zellweger spectrum disorder, a rare peroxisomal disorder that is categorized as a leukodystrophy. As his disease progressed, our journey with Ilan was marked by complex challenges. Ilan passed…
Help Us Bring Hope to Families Like Isabella’s This Holiday Season
I’m the aunt of my beautiful niece, Isabella, who was diagnosed with Vanishing White Matter (VWM) Disease at age four in 2009. Isabella’s joyful spirit and incredible courage inspire me to be the best I can be. She faces the challenges of this devastating condition with a smile that never…
Beverly’s Journey of Hope & Giving Back
“I was so fortunate to have been able to attend the ULF conference without worrying about finances, and other families should not have to be concerned about costs to be there.” Beverly Jones Devoted mother & longtime ULF supporter Beverly Jones, a devoted mother, first connected with the United Leukodystrophy…
In Memorial and Honor: August – September 2024
ULF received donations in memorial or honorarium for the following during the months of August through September 2024. We apologize for any inadvertent omittance or misspellings. Please notify us if we have made a mistake at [email protected] or by calling 815-748-3211 In Honor of Clark Cutler Jennifer Martin-Cutler Saddle up…
Aspa Therapeutics: A Natural History Study of Patients With Canavan Disease
Aspa Therapeutics: A Natural History Study of Patients With Canavan Disease (CANinform Study) [https://clinicaltrials.gov/study/NCT04126005] CANinform is a natural history study of patients with Canavan disease that will help researchers better understand the disease and provide a foundation for Aspa’s gene therapy program. The study is open to individuals with a confirmed diagnosis of Canavan disease.
BridgeBio press release about the RMAT designation for the Aspa clinical trial in Canavan Disease
09.10.2024 at 4:45 PM EDT – Receipt of RMAT Designation is based on preliminary clinical evidence from the CANaspire Phase 1/2 clinical trial, which showed functional improvements in all dosed patients indicating that BBP-812 has potential to address the unmet needs of individuals with Canavan disease – BridgeBio will leverage…
CTX Patient Assistance Program is open and accepting assistance applications
NORD’s Cerebrotendinous Xanthomatosis (CTX) Patient Assistance Program is open and accepting applications for assistance The quickest way to apply is on NORD’s website at: bit.ly/patient-assist. Scroll down to Cerebrotendinous Xanthomatosis (CTX) and click Apply Online.
In Memorial and Honor: May – July 2024
ULF received donations in memorial or honorarium for the following during the months of May through July 2024. We apologize for any inadvertent omittance or misspellings. Please notify us if we have made a mistake at [email protected] or by calling 815-748-3211 In Honor of Dan Mulligan Troy Glendenning Tara Nunez…
An Update from Passage Bio on Out-Licensing of Pediatric Programs to GEMMA Biotherapeutics
On August 1, 2024, Passage Bio shared they entered into a new agreement with GEMMA Biotherapeutics for the out-licensing of three pediatric gene therapy programs. Through this transaction, Passage Bio has granted GEMMA Biotherapeutics the rights for the development and commercialization of PBGM01 for GM1 gangliosidosis, PBKR03 for Krabbe disease,…