Picture this: A desperately ill man with myelodysplastic syndrome in his seventies is fighting for his very life. A beautiful young woman in a foreign country is going about her everyday life.…
Have you ever heard of “ex vivo” gene therapy? If you haven't, it means removing some cells, modifying them, and putting them back in. The goal? Curing the diseases that…
Want some more news, events and announcements on Gaucher disease? We got 'em! [one_half] [/one_half] [one_half_last] Promising Molecule Identified as Possible Treatment for Gaucher, Parkinson's Diseases NICE Changes Mind…
You’ve heard the saying, it takes a village to raise a child? Well, as much as the fiercely independent may chafe at the suggestion, it’s true: raising, teaching, and protecting…
The Neurodegeneration with Brain Iron Accumulation (NBIA) Disorders Association was established in 1996 to serve and support all those affected by PKAN, PLAN, MPAN, BPAN, and CoPAN. Of these, PKAN or…
Bronchopulmonary dysplasia, or BPD, is a chronic lung disease that most commonly occurs in premature infants and is caused by a reaction to being on mechanical ventilation, or from being on…
I think most people experience eye problems at one point or another. Maybe your child came home with "pink eye," and before long, everyone in the household had itchy, burning…
These days Frank drives around to help others with trigeminal neuralgia. He seeks to raise awareness while comforting those who are living with the condition. Before Frank was diagnosed, he…
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