News Archives - United Leukodystrophy Foundation

FDA Approves First Treatment for CTX, a Rare Lipid Storage Disease

News, Research / February 21, 2025 February 21, 2025

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, leading to improper cholesterol breakdown …

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STUDY: The natural history of variable subtypes in pediatric-onset TUBB4A-related leukodystrophy

News, Research / February 21, 2025 February 19, 2025

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 brain responsible for communication between …

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Study: Impaired Peroxisomal Beta-Oxidation in Microglia Triggers Oxidative Stress and Impacts Neurons and Oligodendrocytes

News, Research / February 20, 2025 February 19, 2025

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 down fatty acids — affects …

Study: Impaired Peroxisomal Beta-Oxidation in Microglia Triggers Oxidative Stress and Impacts Neurons and Oligodendrocytes Read More »

28th ASGCT Annual Meeting- Empowering Patients 2025: A Cell and Gene Therapies Summit

News / February 19, 2025 February 19, 2025

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, patients, and caregivers who are …

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Minoxry Therapeutics: Leriglitazone has met the primary endpoint in NEXUS, the pivotal trial for pediatric patients with cALD

Clinical Trials, News / December 13, 2024 December 13, 2024

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 in treated patients, compared to …

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Aspa Therapeutics: A Natural History Study of Patients With Canavan Disease

News / September 24, 2024 September 24, 2024

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

News / September 16, 2024 September 16, 2024

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 the benefits of RMAT designation, …

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CTX Patient Assistance Program is open and accepting assistance applications

News / August 21, 2024 August 21, 2024

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.

An Update from Passage Bio on Out-Licensing of Pediatric Programs to GEMMA Biotherapeutics

News / August 2, 2024 August 2, 2024

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, and PBML04 for metachromatic leukodystrophy. …

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