Over 95% of babies now diagnosed with SMA are treated with Zolgensma gene therapy, per Daniel Grant, vice president for ...

In an interview, Scholar Rock's chief medical officer talks about benefits seen in clinical trials of its muscle-targeted SMA ...

In the last of a series, columnist Sherry Toh shares how she's coping with an intense leg of her journey with chronic pain ...

After facing several damaging storms, columnist Helen Baldwin was delighted to see a daffodil blooming amid the chaos.

Activating a protein called potassium channel Kv2.1 may help improve motor function in SMA, making it a potential SMA ...

Activating a protein called potassium channel Kv2.1 may help improve motor function in SMA, making it a potential SMA ...

A higher dose of Spinraza may be more effective than the currently approved dosing schedule for maintaining motor function in ...

Children with SMA given the gene therapy Zolgensma in infancy are maintaining motor milestones after up to 10 years, new ...