Allan-Herndon-Dudley syndrome (AHDS) is a rare genetic disorder caused by mutations in the MCT8 gene. These mutations result in impaired thyroid hormone transport across the blood-brain barrier. Current treatment options for AHDS are limited and aim to manage symptoms. The first line of treatment involves thyroid hormone replacement therapy with triiodothyronine (T3). T3 supplements help restore thyroid hormone levels in the brain to improve neurological development and function. However, high T3 doses sometimes cause side effects like tremors, hyperactivity, and irregular heartbeat. The other option is thyroxine (T4) therapy which relies on the peripheral conversion of T4 to active T3 within cells. This indirect method of treatment is not very effective as the MCT8 mutations disrupt hormone transportation and activation.
Emerging gene therapy is a promising investigational approach to directly treat the underlying genetic cause of AHDS. In gene therapy, a normal copy of the MCT8 gene would be delivered into the patient's cells and tissues to replace the mutated one. Though still in early research phase, this could correct the hormone transport defect and eliminate the need for lifelong thyroid supplementation. Challenges include developing effective and safe gene delivery vectors and treatments needing to be tailored to individual patients based on their specific MCT8 mutations. More research is underway to validate gene therapy as an innovative treatment for AHDS in the future.
Market Dynamics:
The global Allan-Herndon-Dudley syndrome (AHDS) (MCT8 deficiency) treatment market is mainly driven by the increasing prevalence of AHDS is expected to propel the market growth over the forecast period. For instance, according to the data published by the National Center for Biotechnology Information (NCBI) in January 16, 2020, the identification of more than 160 affected individuals in approximately 15 years suggests that the syndrome is more common than previously thought.
Key features of the study:
Detailed Segmentation:
Global Allan-Herndon-Dudley syndrome (AHDS) (MCT8 deficiency) Treatment Market - Segmentation
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