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. https://geneticliteracyproject.org/2020/01/06/2019-offered-eclectic-mix-of-potentially-breakthrough-treatments-for-genetic-diseases-from-cystic-fibros
blog article
SHARESHARESHARE
2019 OFFERED ‘ECLECTIC’ MIX OF POTENTIALLY BREAKTHROUGH TREATMENTS FOR GENETIC DISEASES, FROM CYSTIC FIBROSIS TO SICKLE CELL
It was a good year for new treatments for genetic diseases! Of the 44 FDA approvals of new drugs, 8 were for 6 single-gene diseases: DMD, beta thalassemia, cystic fibrosis, a form of amyloidosis, and two each for sickle cell disease and porphyria. The eight approvals showcase the diverse therapeutic strategies that are finally leaping from clinical trial to clinical reality. That’s important. DNA, RNA, and protein-based treatments face an especially high bar because of the perceived permanence of a correction at these levels of genetic information. Slowing of disease progression or improvement in one or two symptoms are signs of success, but it might take time for some molecular corrections to translate into fading symptoms. That’s why the multi-pronged strategies are critical. If a gene therapy isn’t leading to rapid or obvious improvement in a child with a brain or muscle disease, then perhaps RNAi, antisense therapy, or enzyme replacement therapy will. Better yet, instead of testing the technologies in tandem, do it in parallel. READ MORE