Abstract:
INTRODUCTION:Lysosomal storage disorders (LSDs) are clinically heterogeneous disorders that result primarily from lysosomal accumulation of macromolecules in various tissues. LSDs are always progressive, and often lead to severe symptoms and premature death. The identification of the underlying genetic and enzymatic defects has prompted the development of various treatment options. AREAS COVERED:To describe the current treatment options for LSDs, the authors provide a focused overview of their pathophysiology. They discuss the current applications and challenges of enzyme-replacement therapy, stem-cell therapy, gene therapy, chaperone therapy and substrate-reduction therapy, as well as future therapeutic prospects. EXPERT OPINION:Over recent decades, considerable progress has been made in the treatment of LSDs and in the outcome of patients. None of the current options are completely curative yet. They are complicated by the difficulty in efficiently targeting all affected tissues (particularly the central nervous system), in reaching sufficiently high enzyme levels in the target tissues, and by their high costs. The pathways leading from the genetic mutation to the clinical symptoms should be further elucidated, as they might prompt the development of new and ultimately curative therapies.
journal_name
Expert Opin Pharmacotherjournal_title
Expert opinion on pharmacotherapyauthors
van Gelder CM,Vollebregt AA,Plug I,van der Ploeg AT,Reuser AJdoi
10.1517/14656566.2012.729039subject
Has Abstractpub_date
2012-11-01 00:00:00pages
2281-99issue
16eissn
1465-6566issn
1744-7666journal_volume
13pub_type
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journal_title:Expert opinion on pharmacotherapy
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abstract:INTRODUCTION:The administration of chemotherapy before (neoadjuvant), rather than after surgery (adjuvant) in early breast cancer has been considered an optional strategy for patients with operable breast cancer. We reviewed this concept considering recent results in the field. AREAS COVERED:Herein, the authors cover ...
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abstract:INTRODUCTION:Docetaxel and paclitaxel are among the most active agents for the treatment of breast cancer. These first-generation taxanes are extremely hydrophobic; therefore, solvents are needed for its parenteral administration. Albumin nanoparticle technology allows for the transportation of such hydrophobic drugs w...
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