A multidisciplinary approach, early diagnosis, and comprehensive caregiver education are crucial in effectively managing ...

This protein is critical for motor neurons to make proteins and grow the "wires" that send signals out to the muscles. When the SMN1 gene is mutated, the body cannot make enough SMN protein ...

The SMN protein localizes to both the cytoplasm and the nuclear bodies. One hallmark of spinal muscular atrophy is the failure of SMN to localize within these nuclear bodies.

Doctors in the US have treated a fetus with a rapidly progressive congenital disease while in the uterus for the very first time.

The scientists found that the child had higher levels of the SMN protein in their bloodstream, compared to babies normally born with the condition. They seemed to have lower levels of nerve damage ...

Evrysdi is designed to treat SMA by increasing and sustaining the production of SMN protein in the central nervous system (CNS) and peripheral tissues, as demonstrated in animal models.

The three diagnostic tests for identifying genetic mutations associated with SMA met new European Union regulatory ...

The doctors found that the infant had higher levels of SMN protein and less nerve damage than other babies born with SMA type 1. In the months since her birth has shown no signs of abnormal muscle ...