Clinical Trial Development Challenges

Rennie McCarthy, Chief Executive Officer at Stealth Biotherapeutics, discusses clinical trial development challenges for ultra-rare diseases.      Stealth Biotherapeutics is developing elamipretide, a mitochondrial protective agent, as a possible treatment...

Treating Primary Immunodeficiency

Lisa Forbes Satter, MD, Associate Professor of Pediatrics at Baylor College of Medicine, explains current treatment options for patients with primary Immunodeficiency (PI).   PI is a heterogeneous group of inherited disorders characterized by impaired immune...

Diagnosing Primary Immunodeficiency 

Lisa Forbes Satter, MD, Associate Professor of Pediatrics at Baylor College of Medicine, explains why it often takes years to properly diagnose Primary Immunodeficiency (PI).   PI is a heterogeneous group of inherited disorders characterized by impaired immune...

Huntington’s Disease Treatment Approval

Rakesh Jain, MD, MPH, Professor, Department of Psychiatry, Texas Tech University School of Medicine – Permian Basin, discusses the recent Huntington’s disease treatment approval.   Newly approved deutetrabenazine (Austedo XR) extended-release tablets...