Trofinetide for the Treatment of Rett Syndrome

Ponni Subbiah, MD, MPH, Senior Vice President Global Head of Medical Affairs and Chief Medical Officer, Acadia Pharmaceuticals, discusses trofinetide for the treatment of Rett syndrome.   Overview of Rett Syndrome Rett syndrome is a neurodevelopmental condition...

Advancements in Cancer Diagnosis

Milan Radovich, PhD, Chief Scientific Officer at Caris Life Sciences, discussed the use of artificial intelligence (AI) to improve the diagnosis of different cancer patients. Each patient’s imaging data, electronic health records, exome sequencing data, etc.,...

Mitochondrial Myopathy

Rennie McCarthy, Chief Executive Officer at Stealth Biotherapeutics, provides an overview of mitochondrial myopathy, or myopathic mitochondrial DNA depletion syndrome.      Mitochondrial myopathy is a rare genetic disorder caused by mutations in nuclear...

What is Sturge-Weber Syndrome?

Karen Ball, founder of the Surge-Weber Foundation discusses Sturge-Weber Syndrome, a rare neurovascular disease.     Sturge-Weber syndrome is a rare neurocutaneous disorder that primarily affects the skin and the central nervous system. It is characterized...