The rare disease pharmaceutical market has been growing rapidly since the Orphan Drug Act in 1983. This article introduces the rare disease treatment market size, market share, and sales performance of pharmaceutical companies leading the market.
Cedars-Sinai scientists have published a study showing that gene therapy may effectively treat Allan-Herndon-Dudley syndrome (AHDS). Researchers administered the treatment to MCT8-deficient mice, and found that cognitive and motor function improved.
As rWGS(rapid Whole Genome Sequencing) has proven to be effective in early diagnosis and reducing costs, the states of California, Michigan, Oregon, Maryland, and Minnesota have made Medicaid cover rWGS testing for sick newborns. However, in order to expand the use of rWGS, the problems of ordering procedures, expensive equipment, and the shortage of qualified experts for analysis must be resolved.
Based on the results of the ‘Next Generation Children’s Project(2019)’ in the UK, research results have been found that rWGS is an effective way to help early diagnosis of sick children compared to conventional genetic testing methods. The neurology clinic had the highest diagnostic rate, and frequent re-analysis also played a significant role in increasing the diagnostic rate.
Rare disease pharmaceutical industry trendsIonis Pharmaceutical and AstraZeneca announced that they had succeeded in Phase 3 clinical trial of co-developing transthyretin amyloidosis treatment(eplontersen). However, the…