In a groundbreaking medical achievement, physicians have administered the world’s first personalized gene-editing therapy to an infant suffering from a rare and fatal genetic disorder. The innovative treatment, described at…
According to a story from globenewswire.com, the gene therapy company Sio Gene Therapies, Inc., has recently released encouraging interim findings from its phase 1/2 clinical trial. This trial is evaluating…
For rare diseases in which no current treatment options are available, such as GM1 gangliosidosis, there is an urgent need to find therapeutics and improve patient outcomes. Gene therapy company…
On July 8, 2021, gene therapy company Lysogene shared that its gene therapy candidate LYS-GM101, for patients with GM1 gangliosidosis, received Fast Track designation from the FDA. Currently, no treatments…
The National Institutes of Health (NIH) held its annual Rare Disease Day event on March 1, 2021 this year. This event showcases groundbreaking research, amazing rare patient stories, and more.…
According to a story from BioSpace, the gene therapy company Axovant Gene Therapies Ltd. recently announced that it has been given Rare Pediatric Disease designation from the US Food and…
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