Buckle up, it's going to be an incredible ride, columnist Helen Baldwin says in a letter to her 13-year-old self.

Many SMA patients continue to see gains in motor function with long-term Spinraza treatment, a real-world study from ...

Meeting your new medical team is never easy, especially when you have a complex and rare disease like SMA, columnist Alyssa ...

It is thought that salbutamol increases production of SMN from the SMN2 gene.Normally, SMN2 produces only about 10% as much functional SMN protein as the SMN1 gene due to a slight difference in the ...