The drug, Ceriliponase alfa from BioMarin, helped in a condition that has led inexorably to disability and death: Batten Disease – specifically CLN2. Families of kids facing this prognosis are…
Happy Friday Patient Worthians! Did you know it was World PI Week this week? We have a post on the role pets have with PI. We also have a story on a…
A special session on CGD will be presented at the 2017 IDF National conference. CGD is a primary immunodeficieny disease and is characterized by skin infections, both fungal and bacterial,…
If you or your loved one is living with idiopathic pulmonary fibrosis (IPF), scientists have proven that there is a link between exercise and longevity—especially for people who have IPF.…
A rare disease that is not so rare: Hormonal causes of high blood pressure have been considered rare but “can be as high as 50% among children and 30% among…
I was at a gala hosted by my local library about two weeks ago. It was a posh affair, as library galas go, and everyone was all dressed up. The…
There’s one person I learned about today who has an admirable life mantra. The young man is D’Marco Jackson, and he lived with aplastic anemia (AA), but he succumbed to…
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