Global ETD Search
Search theses and dissertations gathered from participating repositories worldwide. Every result links back to the library that holds it. No account is needed.
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Showing 1 to 2 of 2 for “"substrate reduction therapy"”.
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No Difference In Health Related Quality of Life Between Therapeutic Options For Type 1 Gaucher Disease
… GD was limited to intravenous enzyme replacement therapy (ERT). ERT reduces symptoms and increases healthrelated quality of life (HRQoL) in people with this condition. In 2014, oral substrate reduction therapy (SRT) was approved for type 1 GD treatment. Although both therapies alleviate disease …
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3D KIDNEY ORGANOIDS AS A PLATFORM FOR INVESTIGATING MONOGENIC DISEASES AND GENE THERAPY APPROACHES
… validating therapies such as Enzyme Replacement Therapy (ERT) and Substrate Reduction Therapy (SRT) for Fabry disease and pharmacological therapy for Lowe syndrome. However, despite being widely adopted in clinics, these therapies have proven to be only partially effective, indicating that new …