Ultragenyx Pharmaceutical Inc

This program provides access to an experimental drug called triheptanoin for patients with Long Chain Fatty Acid Oxidation Disorders. It is for people who have limited treatment options and cannot join a formal clinical trial. The goal is to help manage this rare metabolic condit…

This study will track 140 patients with Glycogen Storage Disease Type Ia for at least 10 years after they received an experimental gene therapy called DTX401. The main goal is to monitor long-term safety and see how well the treatment continues to work over time. Researchers will…

This study is testing an experimental drug called GTX-102 in people with Angelman syndrome, a rare genetic disorder that affects development and causes seizures and movement problems. Researchers want to see if the drug is safe and if it can improve thinking, communication, behav…

This program provides access to the drug Mepsevii for patients with a rare genetic disorder called MPS VII (Sly Syndrome). It is for individuals who have no other treatment options available. The goal is to manage the disease by providing the approved therapy in countries where i…

This study is testing a one-time gene therapy called UX111 for children with Sanfilippo Syndrome Type A, a rare and severe genetic disorder. The therapy involves a single intravenous injection designed to deliver a working copy of a missing gene, with the goal of slowing or stopp…

This study aims to better understand how MPS VII (Sly Syndrome) progresses over time and to gather long-term information on the effectiveness and safety of the treatment vestronidase alfa. It will follow up to 50 patients, both those receiving the treatment and those who are not,…