Therapies for galactosemia: a patent landscape
| العنوان: | Therapies for galactosemia: a patent landscape |
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| المؤلفون: | David J. Timson |
| المصدر: | Pharmaceutical Patent Analyst. 9:45-51 |
| بيانات النشر: | Future Science Ltd, 2020. |
| سنة النشر: | 2020 |
| مصطلحات موضوعية: | Galactosemias, Patents as Topic, Salubrinal, 03 medical and health sciences, chemistry.chemical_compound, 0302 clinical medicine, Cataracts, Aldehyde Reductase, Animals, Humans, Medicine, Transferase, RNA, Messenger, Enzyme Inhibitors, 030304 developmental biology, Genetics, 0303 health sciences, business.industry, Galactosemia, Thiourea, Galactose, Uridylyltransferase activity, General Medicine, medicine.disease, Leloir pathway, chemistry, Cinnamates, Inherited metabolic disease, business, 030217 neurology & neurosurgery |
| الوصف: | Galactosemia is the inherited inability to metabolise galactose. The most common results from a lack of galactose 1-phosphate uridylyltransferase activity. The current treatment, removal of galactose from the diet, is inadequate and often fails to prevent long-term complications. Since 2015, three patents have been filed describing novel therapies. These are: the use of aldose reductase inhibitors to reduce cataracts and, possibly, other symptoms; salubrinal to stimulate cellular stress responses; mRNA therapy to increase cellular galactose 1-phosphate uridylyltransferase activity. The viability of all three is supported by academic studies. The potential and drawbacks of all three are discussed and evaluated. |
| تدمد: | 2046-8962 2046-8954 |
| DOI: | 10.4155/ppa-2020-0004 |
| URL الوصول: | https://explore.openaire.eu/search/publication?articleId=doi_dedup___::6d7a46c6ab5ba00cdb74aecf45701210 https://doi.org/10.4155/ppa-2020-0004 |
| Rights: | OPEN |
| رقم الانضمام: | edsair.doi.dedup.....6d7a46c6ab5ba00cdb74aecf45701210 |
| قاعدة البيانات: | OpenAIRE |
| تدمد: | 20468962 20468954 |
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| DOI: | 10.4155/ppa-2020-0004 |