Spinal muscular atrophy (SMA) is a disorder affecting the motor neurons—nerve cells that control voluntary muscle movement. These cells are located in the spinal cord. Because the muscles cannot ...

One of the Prince and Princess of Wales's first marital homes was the beautiful Anmer Hall in Norfolk. The royal couple, then known as the Duke and Duchess of Cambridge, were gifted the impressive ...

and Prince Louis, who often spend time at their Norfolk residence, Anmer Hall. A sweet clip shared by @norfolkplayparks revealed just how fun it is for little ones. As well as climbing apparatus ...

It’s close enough to London, not too far away." Comic Relief//Getty Images The royal family outside Anmer Hall. A source spoke about Kate and Prince William's decision to move from London back ...

Key to that mission is the “STEER” study, a late-stage trial of children with moderate SMA who are older than two years and have never before received certain targeted medicines like Zolgensma, Biogen ...

A pair of clinical data sets gives Novartis confidence in a new formulation of the company’s gene therapy Zolgensma for the treatment of older patients with spinal muscular atrophy (SMA).

The pivotal trial enrolled only babies under 6 months. Two-year-olds born with the most common and severe form of SMA would likely have lost too many neurons to benefit from the therapy.

OAV101 IT, a version of the gene therapy Zolgensma (onasemnogene abeparvovec-xioi) that’s administered into the spinal canal, can be safely given to people with spinal muscular atrophy (SMA) who were ...