Juvenile Onset Huntington's Disease
The Special Issue “Juvenile Onset Huntington’s Disease” highlights the growing interest in understanding the unique aspects of this ultra-rare disorder. After decades of research, gene therapy trials are underway for Adult Onset Huntington’s Disease (AOHD). However, patients with Juvenile Onset Hunt...
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| Format: | Online |
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| Jezik: | angleščina |
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MDPI - Multidisciplinary Digital Publishing Institute
2021
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| Online dostop: | ONIX_20210501_9783039438112_40 |
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| _version_ | 1869523503325118464 |
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| collection | Directory of Open Access Books |
| description | The Special Issue “Juvenile Onset Huntington’s Disease” highlights the growing interest in understanding the unique aspects of this ultra-rare disorder. After decades of research, gene therapy trials are underway for Adult Onset Huntington’s Disease (AOHD). However, patients with Juvenile Onset Huntington’s Disease (JOHD) are often excluded from these efforts, leaving many questions regarding its phenomenology. The current issue includes seven articles spanning work on the difficult emotional experiences of parents of children with JOHD; a review of the clinical manifestations of JOHD; behavioral issues in JOHD; CAG repeat and age of motor onset; autonomic nervous system dysfunction; and abnormality in MRI metabolic markers. Finally, a review of the therapeutic advances is included, highlighting future possibilities of clinical trials in JOHD subjects. The HD community—patients, family members at-risk for HD, caregivers, health-care professionals and scientists—is keen on expanding our understanding of JOHD. In the flurry of research on AOHD, those with JOHD were seemingly ‘left behind.’ The study of patients who are afflicted early in life with HD has become imperative, with this Special Issue representing just the beginning of the required effort to address this urgent need. |
| format | Online |
| id | doab-20.500.12854ir-68296 |
| institution | Directory of Open Access Books |
| language | eng |
| publishDate | 2021 |
| publishDateRange | 2021 |
| publishDateSort | 2021 |
| publisher | MDPI - Multidisciplinary Digital Publishing Institute |
| publisherStr | MDPI - Multidisciplinary Digital Publishing Institute |
| record_format | ojs |
| spelling | doab-20.500.12854ir-682962024-03-31T13:08:56Z Juvenile Onset Huntington's Disease Nopoulos, Peggy C. Huntington’s disease CAG repeat mutant huntingtin (mHTT) therapeutics neurodegeneration juvenile Huntington’s disease pediatric Huntington’s disease early-onset Huntington’s disease personal experiences caregivers case series juvenile-onset Huntington’s disease T1-Rho neuroimaging behavioral regulation executive function trinucleotide repeat disorder CAG motor onset juvenile-onset Huntington’s Disease autonomic n/a thema EDItEUR::M Medicine and Nursing The Special Issue “Juvenile Onset Huntington’s Disease” highlights the growing interest in understanding the unique aspects of this ultra-rare disorder. After decades of research, gene therapy trials are underway for Adult Onset Huntington’s Disease (AOHD). However, patients with Juvenile Onset Huntington’s Disease (JOHD) are often excluded from these efforts, leaving many questions regarding its phenomenology. The current issue includes seven articles spanning work on the difficult emotional experiences of parents of children with JOHD; a review of the clinical manifestations of JOHD; behavioral issues in JOHD; CAG repeat and age of motor onset; autonomic nervous system dysfunction; and abnormality in MRI metabolic markers. Finally, a review of the therapeutic advances is included, highlighting future possibilities of clinical trials in JOHD subjects. The HD community—patients, family members at-risk for HD, caregivers, health-care professionals and scientists—is keen on expanding our understanding of JOHD. In the flurry of research on AOHD, those with JOHD were seemingly ‘left behind.’ The study of patients who are afflicted early in life with HD has become imperative, with this Special Issue representing just the beginning of the required effort to address this urgent need. 2021-05-01T15:06:24Z 2021-05-01T15:06:24Z 2021 book ONIX_20210501_9783039438112_40 9783039438112 9783039438129 https://directory.doabooks.org/handle/20.500.12854/68296 eng application/octet-stream Attribution 4.0 International https://mdpi.com/books/pdfview/book/3306 https://mdpi.com/books/pdfview/book/3306 MDPI - Multidisciplinary Digital Publishing Institute The Huntington 10.3390/books978-3-03943-812-9 10.3390/books978-3-03943-812-9 46cabcaa-dd94-4bfe-87b4-55023c1b36d0 1c332c1a-850f-45a4-b7c7-56409bcf68bc 9783039438112 9783039438129 102 Basel, Switzerland open access |
| spellingShingle | Huntington’s disease CAG repeat mutant huntingtin (mHTT) therapeutics neurodegeneration juvenile Huntington’s disease pediatric Huntington’s disease early-onset Huntington’s disease personal experiences caregivers case series juvenile-onset Huntington’s disease T1-Rho neuroimaging behavioral regulation executive function trinucleotide repeat disorder CAG motor onset juvenile-onset Huntington’s Disease autonomic n/a thema EDItEUR::M Medicine and Nursing Juvenile Onset Huntington's Disease |
| title | Juvenile Onset Huntington's Disease |
| title_full | Juvenile Onset Huntington's Disease |
| title_fullStr | Juvenile Onset Huntington's Disease |
| title_full_unstemmed | Juvenile Onset Huntington's Disease |
| title_short | Juvenile Onset Huntington's Disease |
| title_sort | juvenile onset huntington s disease |
| topic | Huntington’s disease CAG repeat mutant huntingtin (mHTT) therapeutics neurodegeneration juvenile Huntington’s disease pediatric Huntington’s disease early-onset Huntington’s disease personal experiences caregivers case series juvenile-onset Huntington’s disease T1-Rho neuroimaging behavioral regulation executive function trinucleotide repeat disorder CAG motor onset juvenile-onset Huntington’s Disease autonomic n/a thema EDItEUR::M Medicine and Nursing |
| topic_facet | Huntington’s disease CAG repeat mutant huntingtin (mHTT) therapeutics neurodegeneration juvenile Huntington’s disease pediatric Huntington’s disease early-onset Huntington’s disease personal experiences caregivers case series juvenile-onset Huntington’s disease T1-Rho neuroimaging behavioral regulation executive function trinucleotide repeat disorder CAG motor onset juvenile-onset Huntington’s Disease autonomic n/a thema EDItEUR::M Medicine and Nursing |
| url | ONIX_20210501_9783039438112_40 |