News Archives - United Leukodystrophy Foundation

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

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)

News, Research / By ulfunited

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

News, Research / By ulfunited

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

News, Research / By ulfunited

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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PMD Clinical Study

News / By ulfunited

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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Aspa Therapeutics: A Study of AAV9 Gene Therapy in Children With Canavan Disease

News / By ulfunited

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

News / By ulfunited

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 / By ulfunited

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 / By ulfunited

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 / By ULF Staff

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

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

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

BridgeBio Shares Positive Data from Canavan Disease Study

PMD Clinical Study

Aspa Therapeutics: A Study of AAV9 Gene Therapy in Children With Canavan Disease

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

BridgeBio press release about the RMAT designation for the Aspa clinical trial in Canavan Disease

CTX Patient Assistance Program is open and accepting assistance applications

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

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