1, respectively. Finally, this girl was diagnosed of infantile Sandhoff's disease. After treatment with valproate, levetiracetam combined with an 著录项
Disease progression is rapid, resulting in early death. Currently, there is no curative treatment, with therapy remaining primarily supportive. This case report is of a 13-month-old aboriginal Canadian boy who was referred for further investigations related to global developmental delay and loss of ...
The present disclosure provides methods for the treatment of lysosomal storage disorders using gene replacement therapy. In particular, methods are provided for the treatment of Tay-Sachs disease, Sandhoff Disease, and GM1-gangliosidosis using enzyme replacement therapy. Expression constructs encoding enzymes...
摘要: Focuses on substrate deprivation as treatment for Sandhoof disease, a neurodegenerative disorder. Animals and treatment procedures; Behavioral tests; Horizontal bar test; Biochemical analysis; Histology; Electron microscopy; Symptom onset and life expectancy....
Multi-target-directed triazole derivatives as promising agents for the treatment of Alzheimer's disease. Bioorg Chem. 2019 Jun;87:572-584. doi: https://doi.org/10.1016/j.bioorg.2019.03.058. Epub 2019 Mar 23. PMID: 30928879. 31. Chen SG, Gu RX, Dai H, Wei DQ. Virtual screening for ...
Novel vector design and hexosaminidase variant enabling self-complementary adeno-associated virus for the treatment of Tay–Sachs disease. Hum Gene Ther. 2016;27:509–21. CAS PubMed PubMed Central Google Scholar Osmon KJ, Woodley E, Thompson P, Ong K, Karumuthil-Melethil S, Keimel JG, et...
due to a lack of β-subunits in Sandhoff disease. Because homodimers of β-subunits are more stable than the Hex A heterodimers, some Hex B would be able to assemble despite the presence of the higher concentration of α-subunits in the ER pool. Thus under normal conditions there would be...
IntraBio investigational new drug application approved by the FDA for the treatment of Tay-Sachs and Sandhoff Disease
Disease progression is rapid, resulting in early death. Currently, there is no curative treatment, with therapy remaining primarily supportive. This case report is of a 13-month-old aboriginal Canadian boy who was referred for further investigations related to global developmental delay and loss of ...
Focuses on substrate deprivation as treatment for Sandhoof disease, a neurodegenerative disorder. Animals and treatment procedures; Behavioral tests; Horizontal bar test; Biochemical analysis; Histology; Electron microscopy; Symptom onset and life expectancy.Butters...