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

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

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

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

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 ...

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

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

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 ...

Family caregivers express immense devotion to others, but they should also remember self-care, writes columnist.

Spinraza (nusinersen) may help stabilize lung function in people with SMA types 2 and 3, delaying the need for breathing ...

Combining the experimental and approved SMA therapies BIO101 and Spinraza in the lab was found to improve muscle function in ...