ALEX TLC (Alex, The Leukodystrophy Charity) produces monthly research summaries highlighting recent leukodystrophy research and clinical trials, with a focus on key findings and advancements in treatment. These summaries are prepared by volunteer Biomedical Science students, who provide their interpretations of the latest studies and findings — helping make complex research more accessible to the …
Ionis anounces positive topline results from pivotal study of zilganersen in Alexander disease The Ionis Zilganersen Team shared a message with the Alexander disease community: September 22, 2025 Dear Alexander disease community, Ionis announced today positive initial results from the pivotal clinical trial (NCT: 04849741)1 of zilganersen (ION373) in people with Alexander disease (AxD). In …
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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 …
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 …
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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 …
Aspa Therapeutics: A Study of AAV9 Gene Therapy in Children With Canavan Disease (CANaspire Clinical Trial) [https://treatcanavan.com/canaspire-trial/] Aspa’s investigational gene therapy trial uses an AAV9 vector (adeno-associated virus serotype 9), which is designed to deliver functional copies of the ASPA gene throughout the body, including into the brain, to address the underlying cause of Canavan disease. The AAV9 …
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