Center for Biomedical Engineering and Science, Department of Mechanical Engineering and Engineering Science, University of North Carolina at Charlotte, Charlotte, North Carolina 28223, United States ...
Charcot–Marie–Tooth disease is the most common genetic peripheral neuropathy, with autosomal dominant mutations in Rab7 causing Charcot–Marie–Tooth type 2B disease, which is characterized by the ...
BACKGROUND The lepromatous pole is a stigmatising prototype for patients with leprosy. Generally, these patients have little or no symptoms of peripheral nerve involvement at the time of their ...
Peripheral neuropathy (PN) is a dose-limiting side effect of chemotherapeutic and antibody drug conjugate (ADC) treatment which significantly impacts patients’ quality of life. Despite a high clinical ...
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 ...
1. In this dose-escalation study, using intrathecal gene transfer with scAAV9/JeT-GAN demonstrated potential improvement in motor function. 2. Adverse events related to gene therapy included scoliosis ...
Neurological disorders result in 9.4 million deaths worldwide. Neurofilaments (NFs) are tissue-specific intermediate filaments that maintain neuronal structural stability. Mammalian NFs are composed ...
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 ...
MONDAY, March 25, 2024 (HealthDay News) — Intrathecal gene transfer with scAAV9/JeT-GAN may result in some benefit for children with giant axonal neuropathy, according to a study published in the ...
Treatment for rare childhood disease was well tolerated and slowed loss of motor function. March 25, 2024 – An investigational gene therapy for a rare neurodegenerative disease that begins in early ...
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