A Boston-area biopharmaceutical firm has submitted a biologics license application to the FDA for its investigational, fully ...

A case report described an infant with concurrent HDFN and SMA who was succesfully treated with an onasemnogene abeparvovec infusion.

Columnist Helen Baldwin gains a different perspective following a crushing diagnosis, but glasses weren't what helped her to see clearly.

What is Cushing's syndrome? Cushing's syndrome is a hormonal disorder. It’s caused when you have high levels of the hormone cortisol over a long time. Cushing's syndrome is fairly rare. It most often ...

The kickstand has a new design and can support the Switch 2 from a much lower angle, all the way down to horizontal. Handy! Image credit: Nintendo Onto the Switch 2 Joy-Con, which have been given ...

A combination of a ready-to-use SMN protein and an experimental treatment targeting the SMN2 gene, which works in a similar way to Spinraza (nusinersen), eased abnormalities in motor neurons derived ...