An HIV-derived nucleoside therapy now treats rare genetic diseases by restoring mitochondrial DNA and improving muscle ...

British singer Jesy Nelson recently took to Instagram to share a devastating update about her 8-month-old twin daughters.

A new gene therapy treatment for Duchenne muscular dystrophy (DMD) shows promise of not only arresting the decline of the muscles of those affected by this inherited genetic disease, but perhaps, in ...

Former Little Mix star breaks down as she explains eight-month-old Ocean and Story have severe SMA and may never walk ...