The news that GlaxoSmithKline would divest from rare diseases intrigued industry watchers and left employees at the UK's largest pharmaceutical worried about their livelihoods. But, for patient advocates like myself who had watched GSK become a global leader in the three decade long effort to pioneer gene replacement therapies, the news was devastating. One of the treatments GSK will sell is a gene therapy for metachromatic leukodystrophy (MLD). My daughter Cal, who is now 7

It was a pleasure to be part of Cal’s Cupcake Challenge and experience the immense impact that Cal, supported by her family and community, has had on raising awareness and support for leukodystrophy. As future nurses, we are dedicated to supporting our patients while they fight against illnesses. This event taught us that our duty, as nurses, is not only to provide direct medical care for our patients; we are also called to join the families of our patients in an effort to ad