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

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

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

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 ...

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

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

In episode 154, host Kevin Schaefer talks with Coach Damon Vincent from Lafayette, Louisiana. Damon is an adaptive fitness specialist who works with clients with various rare diseases and disabilities ...

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

During this season of Thanksgiving, columnist Halsey Blocher reminds readers that they don't have to give a lot to make an impact.

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 ...