Month: October 2024

Experiences of mothers of long-term surviving patients with cerebral adrenoleukodystrophy: a qualitative study

Research / November 25, 2024

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 …

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Gene therapy shows long-term benefit for patients with Cerebral adrenoleukodystrophy (CALD)

Research / November 25, 2024

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 …

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Viking Therapeutics Announces Results from Phase 1b Clinical Trial of VK0214 in Patients with X-ALD

Clinical Trials / November 28, 2024

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 Canavan Disease Study

Clinical Trials / November 25, 2024

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 …

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In Memorial and Honor: August – September 2024

In Memory or In Honor Donation / October 15, 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 – Kicks for clark event. …

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PMD Clinical Study

Clinical Trials / November 25, 2024

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 …

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