Children with SMA in the U.K. can switch treatments based on practical needs and preferences without unexpected disease ...

Gene therapy treatment gives Baby Kiama a lifeline, saving him from world’s most expensive drug that he needed to slow down a ...

Since 2016, the FDA has approved three disease-modifying treatments for spinal muscular atrophy, with several ...

OAV101 IT is an investigational gene replacement therapy designed to directly address the genetic root cause of the disease by replacing the nonworking SMN1 gene with a single dose. In the ...

A blood glucose test measures how much sugar you have in your blood. You may have a blood glucose test in a doctor’s office, laboratory, or with a fingerstick meter at home. When you eat ...

SMA, a rare inherited neuromuscular disease, is mainly caused by mutations in the SMN1 gene. These mutations lead to a lack of the SMN protein that’s important for the health of the specialized nerve ...