Yesterday Ultragenyx Pharmaceutical provided a Phase I/II update for its investigational antisense oligonucleotide GTX-102, demonstrating substantial symptomatic improvement in patients with the rare genetic disorder Angelman syndrome.
Tag Archive for: genetic disorder
The drug candidate, olezarsen, showed statistically significant reduction in levels of triglyceride compared to placebo, as well as showed a 100% reduction in inflammation of the pancreas.
The deal is the first large acquisition under new CEO Christopher Viehbacher as he seeks to return the drugmaker to growth.
Despite beating analyst expectations, Takeda sustains a mid-stage defeat in a rare genetic disorder, leaving the fate of an investigational enzyme replacement therapy uncertain.
After an initial rejection, BioMarin has finally secured the FDA’s approval for Roctavian, the first gene therapy in the U.S. for the most common form of the bleeding disorder.
The recommendation follows a review by EMA’s human medicines committee (CHMP) that concluded the benefits of the drug did not outweigh risks, according to the regulator.
For $150 million upfront, Sanofi will gain access to Maze’s lead asset MZE001, which inhibits the GYS1 protein in the rare genetic disorder.
The U.S. Food and Drug Administration (FDA) has approved Acer Therapeutics Inc.’s drug for treating patients with a type of genetic disorder called urea cycle disorders (UCDs), according to the health regulator’s website.
The company delayed the timeline for reporting key data from a trial of its cannabis-based drug to treat a rare genetic disorder, citing difficulties in enrolling patients amid a rise in respiratory illnesses.
