A friend's 2-year old daughter who was recently diagnosed with Metachromatic Leukodystrophy (MLD). I feel terribly sorry for this poor little girl and her heartbroken parents.
I heard there is an investigation going on in the US using Metazym for the disease, same as the one in denmark, but no chance for her to go on the trial in Denmark.
Please could any of you find out any information about the study in the US? The parents are willing to give everything just for any teeny tiny hopes for their lovely daughter.
Thank you very much for any help in advace.
Thank you.