
Innovative Research in Neurological Diseases Highlighted for Rare Disease Day 2025
Neurolixis is proud to showcase its groundbreaking research and development for rare neurological conditions in honor of Rare Disease Day, on February 28, 2025.
๐ฆ๐ฝ๐ถ๐ป๐ผ๐ฐ๐ฒ๐ฟ๐ฒ๐ฏ๐ฒ๐น๐น๐ฎ๐ฟ ๐๐๐ฎ๐ ๐ถ๐ฎ (๐ฆ๐๐)
Spinocerebellar Ataxia is a group of inherited neurodegenerative disorders that lead to loss of motor control, affecting coordination and balance. Neurolixis is developing ๐ก๐๐ซ-๐ญ๐ญ๐ฎ to alleviate motor symptoms, protect neurons, and slow the progression of the disease. Preclinical and early clinical results have shown promising improvements in motor function and stabilization of disease symptoms, offering new hope for individuals living with SCA.
๐๐ฟ๐ฎ๐ด๐ถ๐น๐ฒ ๐ซ ๐ฆ๐๐ป๐ฑ๐ฟ๐ผ๐บ๐ฒ (๐๐ซ๐ฆ)
Fragile X Syndrome is the most common genetic cause of autism and intellectual disabilities. Neurolixis is developing ๐ก๐๐ซ-๐ญ๐ฌ๐ญ, a drug candidate that improves mood and cognition and shows disease-modifying neuroplastic activity in transgenic FXS mice. This suggests that it may alleviate core symptoms and enhance cognitive development in children affected by Fragile X Syndrome.
๐ฅ๐ฒ๐๐ ๐ฆ๐๐ป๐ฑ๐ฟ๐ผ๐บ๐ฒ
Rett Syndrome primarily affects young girls and leads to severe cognitive and motor impairments. The Neurolixis drug candidate, ๐ก๐๐ซ-๐ญ๐ฌ๐ญ, shows promising activity in transgenic Rett mice, improving their cognition and respiratory function and offering a potential path to clinical benefit for individuals living with Rett syndrome.
๐๐ฏ๐ผ๐๐ ๐ก๐ฒ๐๐ฟ๐ผ๐น๐ถ๐ ๐ถ๐
Neurolixis specializes in the discovery and development of novel therapies for rare and neurological diseases. The Neurolixis drug candidates selectively target specific brain regions to modulate serotonin neuron activity. This approach aims to enhance therapeutic efficacy while minimizing side effects. The company's pipeline includes NLX-112 (Phase 2), which targets motor disorders, NLX-101 (Phase 1) for treating rare autism spectrum disorders, including Fragile X and Rett syndromes, and NLX-204, a preclinical candidate that shows potential as a rapid-acting antidepressant and analgesic, utilizing a non-opioid mechanism.
๐ Information on Rare Disease Day: https://www.rarediseaseday.org/
๐ 3-minute video summary of the Neurolixis rare disease programs: https://t.ly/SNq4H
๐ Pharmacology review of Neurolixis serotonin 'biased agonists': https://t.ly/hCB8y
ADRIAN NEWMAN-TANCREDI
NEUROLIXIS
contact@neurolixis.com
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