Soleno Therapeutics has announced that they will be hosting a Key Opinion Leader (KOL) webinar on Thursday, February 4 at noon EST. This webinar will focus on Prader-Willi syndrome, specifically the unmet medical need that affected individuals face and the development of Soleno’s DCCR tablet.
About Prader-Willi Syndrome
Prader-Willi syndrome (PWS) is a rare, genetic disorder that is characterized by a constant sense of hunger (hyperphagia). This hunger begins at age two. Physical, mental, and behavioral problems are all common in those with this syndrome. People may experience intellectual impairments and learning disabilities. An error in one or more of the genes on chromosome 15 causes this condition. These errors disrupt the function of the hypothalamus, which is the part of the brain that is responsible for releasing hormones. It is typically a sporadic mutation that leads to the errors on chromosome 15.
Symptoms of Prader-Willi syndrome will change as people age. Right after birth, symptoms include poor muscle tone, distinctive facial features like a narrow forehead, almond-shaped eyes, and a triangular mouth; poor sucking reflex, poor responsiveness, and underdeveloped genitals. As a child reaches early childhood, symptoms may change to hyperphagia, poor growth and physical development, cognitive impairment, delayed motor impairment, obesity, speech and behavioral problems, short stature, small hands and feet, and sleep disorders. Treatment is symptomatic, and it includes good nutrition, human growth hormones, weight management, sex hormone treatment, behavioral and physical therapy, and sleep disorder treatment.
About the Webinar
This webinar focuses on the treatment options for Prader-Willi syndrome patients, and it will also provide an update on the development of Soleno’s Diazoxide Choline Controlled Release (DCCR) tablet, which is taken once a day. Experts will be leading the event: Dr. Theresa Strong from the Foundation for Prader-Willi Research and Dr. Jennifer Miller from the University of Florida.
Dr. Miller leads the Prader-Willi syndrome program at the University of Florida, meaning that she is connected to the largest cohorts of patients throughout the world. She holds a background in pediatrics and pediatric endocrinology, along with clinical investigation.
Dr. Strong is the director and co-founder of the Foundation for Prader-Willi Research, which was created to enhance the treatment, understanding, and awareness for PWS. She holds a doctorate in medical genetics and has previously worked on gene therapies for cancer.