Santhera has completed a rolling application for its Duchenne muscular dystrophy (DMD) therapy vamorolone in the US, setting up a possible approval and launch in the latter half of 2023.
Being diagnosed with a rare disease is often a bolt from the blue, and while each person’s journey is different, there are common challenges that bind patient communities together.
When he was first told he had LHON, a rare genetic disease that would rob him of his sight, James Ferguson’s first thought was that it would also rob him of his identity.
