Treatment with Spinraza (nusinersen) can help stabilize or improve motor function in adults with SMA, according to a new ...

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.

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

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 ...

This weekend kicked off the MDA annual Clinical & Scientific Conference, bringing together the neuromuscular disease ...

Data from the SAPPHIRE study formed the basis of Scholar Rock's U.S. Food and Drug Administration request to approve ...

Unearthing an old stuffed animal led columnist Alyssa Silva to recall the memory of realizing how different her childhood was ...