FDA Approves Trofinetide to Treat Rett Syndrome

The US Food and Drug Administration (FDA) has approved trofinetide (Daybue) to treat persons with Rett syndrome who are two years of age and older. This the first and only drug approved for the treatment of Rett syndrome. ​​Rett syndrome is a rare progressive...

Diagnosing Rett Syndrome

  Jana von Hehn, PhD, Chief Scientific Officer of the Rett Syndrome Research Trust, discusses the challenges of diagnosing Rett syndrome. Rett syndrome is a multisystem disorder that primarily affects girls. Only in rare cases are boys affected (who may...

Myasthenia Gravis : A History Lesson

  Henry Kaminski, MD, Professor of Medicine at The George Washington University ,provides a brief history lesson on our understanding of myasthenia gravis (MG). MG is a chronic autoimmune neuromuscular disease. Common symptoms include weakness of the muscles that...