Nearly 4 of every 5 infants with spinal muscular atrophy (SMA) type 1 born in Italy during the era of disease-modifying therapies (DMTs) are still alive, illustrating that such treatment has ...
Have you ever dreamed of being a princess (or a prince)? I think most of us have, adults included. I’ve always been a Disney fan, and it has no shortage of princesses to choose from. Each one is ...
Nearly 4 of every 5 infants with spinal muscular atrophy (SMA) type 1 born in Italy during the era of disease-modifying therapies (DMTs) are still alive, illustrating that such treatment has ...
Natalie Katz, MD, is an assistant professor of pediatrics in the division of neurology at Duke University and co-directs the Duke Children’s Neuromuscular Program, which provides comprehensive ...
As I reflect on 2024, I see that some great things have happened for me. I’m trying to figure out how to multiply those great things so they happen more often. This is one way I can gain an advantage ...
Edward Smith, MD, is an adjunct professor of pediatrics at Duke University Hospital. He earned his medical degree from the University of Mississippi School of Medicine and has practiced since 2007. He ...
Spinal muscular atrophy (SMA) is a rare genetic condition characterized by progressive muscle weakness and atrophy. It mainly affects motor function, but often also causes problems with speaking, ...