Niemann-Pick UK actively works to raise awareness about the harmful stigma associated with rare diseases and disabilities, as well as supporting collaborative research, and believe that the patient and community voice is essential to the successful development of new therapies
LDA Research consultant, Andrew Grant, highlights the shared common experiences of rare disease patients discussed in our Rare Together podcasts
Today, Dr. David Fajgenbaum celebrates being in remission for ten and a half years from the rare Castleman disease. LDA Research’s director Lucy Doorbar talks to him about how he discovered a treatment for his disease, and the importance for pharmaceutical companies to understand the patient experience.
As many as 200 million children globally have a rare disease, yet most aren’t diagnosed for years after birth. Instead, parents face an array of difficult symptoms often dismissed by doctors as “typical” developmental delays. In honor of FOXG1 Syndrome, Awareness Month this November, we’ll look at this disease as an example. I’m the mother to a little boy with FOXG1 Syndrome, and I, too, was once dismissed by doctors—to the point where I felt powerless and alone, with nowhere to turn to help my sick baby.
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