Journal of Clinical InvestigationTautz, L. (2015). PTP1B: a new therapeutic target for Rett syndrome. J. Clin. Invest. 125, 2931-2934. doi: 10.1172/JCI83192Tautz L. PTP1B: a new therapeutic target for Rett syndrome. J Clin Invest 2015;125:2931-4....
of new therapeutics for Rett syndrome Helen Leonard1* , Wendy Gold2, Rodney Samaco3, Mustafa Sahin4, Timothy Benke5 and Jenny Downs1 Abstract Rett syndrome is associated with severe functional impairments and many comorbidities, each in urgent need of treatments. ...
In 2018, Acadia entered into an exclusive license agreement with Neuren Pharmaceuticals Limited (ASX: NEU) for the development and commercialization of trofinetide for the treatment of Rett syndrome and other indications in North Am...
Scientists have identified how Rett syndrome progresses at the molecular level. The loss of MeCP2 in adult brains triggers rapid gene dysregulation, independent of developmental factors. These findings highlight a critical early window for potential therapeutic intervention. Credit: SciTechDaily.com Sci...
. Epcoritamab has been approved for relapsed or refractory diffuse large B cell lymphoma and high-grade B cell lymphoma after at least two prior systemic treatments via priority review and accelerated approval (Frampton2023). The most common treatment-related AE include cytokine release syndrome, ...
Testing these various potential treatments in humans may help us understand the indications that an approach will—or will not—translate across species. A similar story of potential treatment is emerging in Rett syndrome, where X-linked dominant loss of MECP2 gene function leads to regression in...
Anavex Life Sciences Corp. (Nasdaq: AVXL) is a person-focused, biopharmaceutical company dedicated to therapeutic discovery and development of targeted CNS treatments.
--Anavex Life Sciences Corp., a clinical-stage biopharmaceutical company focused on developing innovative treatments for Alzheimer's disease, Parkinson's disease, schizophrenia, neurodevelopmental, neurodegenerative, and rare diseases, including Rett syn
The company’s leading drug candidate, ANAVEX 2-73 (blarcamesine), has successfully completed Phase 2a and Phase 2b/3 clinical trials for Alzheimer’s treatment, a Phase 2 study in Parkinson’s disease dementia, and Phase 2/3 trials for adult and pediatric patients with Rett syndrome. ...
Disorders of the central nervous system (CNS), in particular those leading to cognitive deficits, are the main challenge facing medicine in the 21st century. Pathophysiologically based cures of CNS disorders do not yet exist; at best contemporary medicine is limited to symptomatic treatments. This ...