According to a story from PR Newswire, the pharmaceutical company Biohaven announced that they have received clearance from the Food and Drug Administration (FDA) to begin work on its clinical trials for BHV-5000. People have already begun enrolling for the first trial, which will test the effects of both single and multiple dose regimens on healthy volunteers. The trial will specifically focus on the processing of the medication in the body and assess safety and potential side effects. BHV-5000 is under development as a potential treatment for Rett syndrome.
Rett syndrome is a brain disorder caused by a genetic mutation. The syndrome only appears in women, usually with the first symptoms appearing about six months after birth. Symptoms include slowed or reduced growth, small head size, and issues with walking and movement. Other issues include difficulty sleeping, seizures, and scoliosis. Males who have the mutation rarely survive the term of the pregnancy. There is no cure. Most people with Rett syndrome live into middle age, and the condition can progress through several different stages as the person develops and ages. The syndrome also have several variants that are classified based on the symptoms that are most prominent. Although the syndrome can be inherited from a parent, in some cases it is caused by a sporadic mutation that occurs in the individual. To learn more about this syndrome, click here.
Currently, there are no treatments available that are specifically meant to treat Rett syndrome. BHV-5000 is specifically hoping to address some of the breathing irregularities that are often present with Rett syndrome. These can cause significant problems, particularly in young children. If the initial trial proceeds without any major issues, then Biohaven will move forward with clinical trials using Rett syndrome patients.
