NSAID Diflunisal Slows Neurodegenerative Progression Early axonal dysfunction may be detected prior to the development of clinical symptoms of diabetic neuropathy. (HealthDay News) – Early axonal ...
Research group from the University of Helsinki, Finland, has identified a new disease gene for early-onset axonal neuropathy and mild intellectual disability through an international research network, ...
Giant axonal neuropathy (GAN) is a rare, autosomal recessive neurodegenerative disorder, characterized by a spectrum of symptoms and a challenging prognosis. Now, a study published recently in the New ...
University of North Carolina (UNC) School of Medicine researchers have developed an innovative, experimental gene transfer-based treatment for children with giant axonal neuropathy (GAN). Researchers ...
DALLAS, Jan. 31, 2023 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric, clinical -stage gene therapy company focused on developing and commercializing AAV-based gene ...
Hannah’s Hope is a charity that was formed in 2008 after then-Plattsburgh residents Lori and Matt Sames’ daughter was diagnosed with a rare progressive nerve disease. Known informally as GAN, the ...
GeneDx’s Hereditary Neuropathy Panel will be available free of charge to individuals at risk for or suspected of having GAN Taysha will collaborate with Hereditary Neuropathy Foundation and ...
Biomarkers are generally used as diagnostic and prognostic tools in clinical practice, but are not yet available for neuropathies. Studies now suggest that neural protein levels in serum and ...
Giant axonal neuropathy is an inherited condition involving dysfunction of a specific type of protein in nerve cells (neurons). The condition is characterized by abnormally large and dysfunctional ...
In a recent study of 33 families with the inherited disorder axonal neuropathy with neuromyotonia, researchers identified eight loss-of-function mutations in the HINT1 gene, which encodes histidine ...
"Alterations in axonal membrane and channel function occur early in the disease course, prior to discernable large fibre involvement, and has the potential to be used as an early biomarker of ...
Efficacy data for high dose cohort demonstrated clinically meaningful and statistically significant improvement in MFM32 by Year 1 compared to natural history (n=3) Long-term durability data across ...