Strategies to combat Tay-Sachs disease - ScienceDirect
Por um escritor misterioso
Last updated 21 março 2025


Miglustat in late-onset Tay-Sachs disease: a 12-month, randomized, controlled clinical study with 24 months of extended treatment - Genetics in Medicine

Mitochondrial Dysfunction and Neurodegeneration in Lysosomal Storage Disorders: Trends in Molecular Medicine

Long-Term Correction of Sandhoff Disease Following Intravenous Delivery of rAAV9 to Mouse Neonates: Molecular Therapy

Current treatment options and novel nanotechnology-driven enzyme replacement strategies for lysosomal storage disorders - ScienceDirect

Tay-Sachs disease - Genes and Disease - NCBI Bookshelf

Infantile gangliosidoses: Mapping a timeline of clinical changes - ScienceDirect

Infantile gangliosidoses: Mapping a timeline of clinical changes - ScienceDirect

The first family with Tay-Sachs disease in Cyprus: Genetic analysis reveals a nonsense (c.78G>A) and a silent (c.1305C>T) mutation and allows preimplantation genetic diagnosis - ScienceDirect

Natural history of Tay-Sachs disease in sheep - ScienceDirect

Late-onset Tay-Sachs disease: Phenotypic characterization and genotypic correlations in 21 affected patients - Genetics in Medicine

Tay-Sachs disease in Jacob sheep - ScienceDirect
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