News

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

Clinical Trials, News / 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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Fall 2024 Newsletter

News / November 25, 2024

The Fall 2024 ULF Newsletter has been posted! Find it here

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

News / 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

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

News / 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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Vigil Neuro: ALSP Community Letter

News / November 17, 2023

The ULF is happy to share a community letter from Vigil regarding their potential treatment option for those affected by ALSP! Here is a preview but click the links below to read the whole letter and press release.  “Dear ALSP Community Members: This week we announced positive interim data from IGNITE, our Phase 2 clinical …

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ALSP Community Survey: Genetic Testing

News / October 31, 2023

Vigil Neuroscience, a biotechnology company researching potential treatments for ALSP, is engaging with the ALSP patient community, caregivers, advocacy groups, and healthcare providers to better understand the needs of community members.  Open Health is an independent company engaged by Vigil Neuroscience to conduct a survey to understand behaviors and attitudes towards genetic testing for ALSP in the community.  Results from …

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