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 ...

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 ...

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 ...

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 ...

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 ...

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 ...

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 ...

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 ...

"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 ...

(RTTNews) - Clinical-stage gene therapy company Taysha Gene Therapies, Inc. (TSHA), Tuesday announced that the company will discontinue the development of its TSHA-120 program in evaluation for the ...

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 ...

A new Guillain-Barré syndrome mimic that appeared to be triggered by mild infection emerged in children with biallelic RCC1 variants, a case series showed. One of the first cases was seen in a ...