Spinal muscular atrophy with respiratory distress (SMARD) is a rare genetic condition that typically affects infants and children. It causes muscle weakness and breathing problems. Spinal muscular ...

Huntington's disease is caused by an inherited genetic change that causes production of an altered protein. This leads to nerve damage in areas of the brain that control movement and thinking.

A gene therapy candidate extended survival in a mouse model of SMARD1, supporting its testing in a clinical trial for this rare form of SMA.

The tiny zebrafish is helping researchers rapidly determine whether a newborn's genetic mutation is likely to cause spinal ...