Disease models for the development of therapies for lysosomal storage diseases
نویسندگان
چکیده
منابع مشابه
Emerging therapies and therapeutic concepts for lysosomal storage diseases
Introduction: The success of the first enzyme replacement therapy (ERT) for a lysosomal storage disease (LSD) and the regulatory and commercial incentives provided by authorities for orphan and rare diseases has spawned a massive interest for developing drugs for these intriguing but devastating genetic disorders. The potential for new drugs in this arena is vast, as not only a high number of L...
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15 صفحه اولJuvenile Gm2 Gangliosidosis: a Model for Investigation of Small-molecule Therapies for Lysosomal Storage Diseases
Juvenile GM2 gangliosidosis (jGM2) is a group of inherited neurodegenerative diseases caused by deficiency of lysosomal β-hexosaminidase A (Hex A) resulting in GM2 ganglioside accumulation in brain. Like many other lysosomal storage diseases (LSDs), no specific treatment currently exists. In order to establish clinical outcomes for the investigation of potential therapies for jGM2, I collected ...
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Enzyme replacement therapy for lysosomal storage diseases.
Enzyme replacement therapy (ERT) has been approved for 6 lysosomal storage diseases (LSDs) worldwide including Japan. These diseases include Gaucher disease (GD), Fabry disease, mucopolysaccharidosis (MPS) types I, II, and VI, and Pompe disease (PD). The efficacy and safety of ERT for LSDs has been confirmed by extensive clinical trials. However, there are still obstacles to successful ERT, suc...
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ژورنال
عنوان ژورنال: Annals of the New York Academy of Sciences
سال: 2016
ISSN: 0077-8923
DOI: 10.1111/nyas.13052