Columnist Helen Baldwin finds the perfect gift for her granddaughter's birthday, one of the many unexpected blessings in her ...

SMA gene therapy Zolgensma safely improved motor function in children with SMA who needed tracheostomy, a real-world study showed.

Columnist Connie Chandler writes about her family's tradition of baking and decorating holiday cookies, a project that SMA ...

Fatigue in the diaphragm may contribute to breathing issues in people with spinal muscular atrophy, a new study reports.

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

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

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 ...

The FDA's approval of SMA treatment Itvisma marks an "important moment" for the SMA community, a Novartis executive says.

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

Rio Landa, physician whose 14-year-old son, Mateo, is diagnosed with spinal muscular atrophy (SMA) type 2, describes her approach on seeking second opinions or voicing concerns about changes in SMA ...

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

The gene therapy Zolgensma (onasemnogene abeparvovec-xioi) has been newly approved to treat patients with spinal muscular atrophy (SMA) through intravenous (IV) delivery, meaning systemically. However ...