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 fades, and she brings happiness …
Help Us Bring Hope to Families Like Isabella’s This Holiday Season Read More »
“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 Foundation (ULF) after her young …
The study publicshed in the New England Journal of Medicine titled “Hematologic Cancer after Gene Therapy for Cerebral Adrenoleukodystrophy” investigates the long-term safety of the gene therapy elivaldogene autotemcel (eli-cel) in patients with cerebral adrenoleukodystrophy (CALD). The therapy modifies patients’ own blood-forming stem cells to include a functional gene to manage CALD. However, 7 of …
Hematologic Cancer after Gene Therapy for Cerebral Adrenoleukodystrophy Read More »
The Fall 2024 ULF Newsletter has been posted! Find it here
Experiences of mothers of long-term surviving patients with cerebral adrenoleukodystrophy: a qualitative study Overview: The study focuses on the lived experiences of mothers caring for long-term survivors of cerebral adrenoleukodystrophy (CALD), a rare genetic disorder affecting the nervous system. It employs qualitative research methods, including in-depth interviews with mothers, to gather rich, personal insights. Key …
Gene therapy shows long-term benefit for patients with Cerebral adrenoleukodystrophy (CALD) Overview: In a study published in The New England Journal of Medicine, lentiviral gene therapy using elivaldogene autotemcel (eli-cel) showed promising results in treating cerebral adrenoleukodystrophy (CALD) in boys. Over a 24-month period, 91% of participants survived without major functional disabilities, and 94% showed …
Reductions in Very Long-Chain Fatty Acids and Plasma Lipid Levels Observed After 28 Days of Once Daily Dosing
VK0214 Shown to be Safe and Well-Tolerated in 28-Day Study
SAN DIEGO, Oct. 9, 2024 /PRNewswire/ — Viking Therapeutics, Inc. (Viking) (NASDAQ: VKTX), a clinical-stage biopharmaceutical company focused on the development of novel therapies for metabolic and endocrine disorders, today announced positive data from the company’s Phase 1b clinical trial of VK0214, a novel small molecule agonist of the thyroid hormone receptor beta (TRβ), in patients with X-linked adrenoleukodystrophy (X-ALD). Results from this study showed VK0214 to be safe and well-tolerated following once-daily dosing over the 28-day study period. In addition, significant reductions were observed in plasma levels of very long-chain fatty acids (VLCFAs) and other lipids, as compared to placebo.
BridgeBio Shares Positive Data from High Dose Cohort of Phase 1/2 CANaspire Study of Gene Therapy BBP-812 for Canavan Disease at ESGCT 2024 –Continued, progressive improvement in motor function and achievement of motor milestones at 12-months post-treatment represents an important and statistically significant change, in contrast to the disease course observed in BridgeBio’s ongoing CANinform …
BridgeBio Shares Positive Data from Canavan Disease Study Read More »
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 – Kicks for clark event. …
Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD) Study Overview Brief Summary The primary purpose of this study is to evaluate the safety and tolerability of ION356. Detailed Description This is a Phase 1b, open-label multiple-ascending dose (MAD) study of ION356 …
