Healio15h
FDA receives biologics license application for apitegromab in spinal muscular atrophy
A Boston-area biopharmaceutical firm has submitted a biologics license application to the FDA for its investigational, fully ...
hdfncompanion3d
Infant with HDFN and spinal muscular atrophy successfully treated
A case report described an infant with concurrent HDFN and SMA who was succesfully treated with an onasemnogene abeparvovec infusion.
Johns Hopkins Medicine10d
Cushing's Syndrome
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 ...
SMA27d
Raising SMN protein levels may be helpful early step in SMA treatment
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 ...