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 ...

When columnist Ari Anderson moved upstairs for a few days, it required a lot of logistical planning. Seemingly small changes ...

SMA expert Brian Lin, PhD, shares his thoughts on aging with SMA, treatment, advocacy, and supporting patients across their ...

SMA expert Brian Lin, PhD, shares his thoughts on aging with SMA, treatment, advocacy, and supporting patients across their ...

Like one of her favorite poems describes, columnist Brianna Albers always feels like she's digging herself out of a new hole in her life.

After a meeting with the FDA, Scholar Rock is planning a 2026 resubmission of its application seeking approval of apitegromab ...