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

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

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

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 ...

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 ...

The SMA gene therapy candidate OAV101 IT, a version of Zolgensma, can be safely given to previously treated patients, per new ...

Columnist Sherry Toh thanks the dedicated nurses who've attended to her at various hospitals and at home over the years.

Columnist Ari Anderson reflects on the memories that made his old house so special and looks forward to making many more in ...