13.03.24
06.03.24
Yesterday, we were greatly disappointed to learn that the UK's National Institute for Health and Care Excellence (NICE) has decided not to recommend Sanofi's Xenpozyme (olipudase alfa) as a treatment for acid sphingomyelinase deficiency (ASMD; Niemann-Pick Disease) in people with type AB or type B...
Read more08.02.24
We're pleased to announce this year's Regional Family Days, designed to bring our community together for support and solidarity. Here's the schedule...
Read more07.02.24
02.02.24