The specialty pharmacy Orsini was selected as a partner to provide Itvisma, the newly approved gene therapy for people with ...

Columnist Connie Chandler gives thanks for the acts of kindness her caregivers perform — the sweet stuff that makes life so ...

Columnist Sherry Toh prefers holiday giving rather than receiving and making others happy, because what she wants isn't found in stores.

Guest writer Kelly Bertsch initially felt hopeless after her son Luke's SMA diagnosis, but she is now watching his bright ...

A high-dose regimen of nusinersen (Spinraza) has been recommended for approval by the CHMP, marking a potential new option ...

The U.S. Food and Drug Administration has approved Itvisma as the first gene therapy for spinal muscular atrophy patients ...

There are several treatment options available for spinal muscular atrophy (SMA), a rare genetic condition characterized by progressive muscle weakness and wasting. The disease mainly affects motor ...

Columnist Sherry Toh reflects on winning her second achievement award this year and thanks all those who supported her along ...

Kelly Bertsch is the mother of Luke Bertsch, 15, who lives with spinal muscular atrophy type 2. Luke was diagnosed just ...

After a “constructive and collaborative in-person” meeting with the U.S. Food and Drug Administration (FDA), Scholar Rock is now planning to once again seek approval of its muscle-strengthening agent ...

Mutations that cause spinal muscular atrophy (SMA) may trigger problems with how bone cells communicate, leading to abnormally weakened bones, according to a study done in mice and cells. The findings ...

Treatment with Spinraza (nusinersen) may help stabilize lung function in people with spinal muscular atrophy (SMA) types 2 and 3, a study suggested. “Treatment of SMA types 2 and 3 with nusinersen may ...