Penn Medicine's Orphan Disease Center will host its fifth annual Million Dollar Bike Ride, where cycling teams that raise money for
rare disease research challenge themselves to ride 13, 34, or 72 miles across the Philadelphia area.
Not exact matches
In new
research, scientists reprogrammed skin cells from patients with
rare blood disorders into iPSCs, highlighting the great promise of these cells in advancing understanding of those
challenging diseases — and eventually in treating them.
Results of the study, which earlier this year won first prize in the inaugural «PAN
Challenge» for
research on improving access to critical medications for Americans with chronic and
rare diseases, showed that patients in the latter, high - cost group were significantly less likely (45.3 percent vs. 66.9 percent) to have a Part D claim for a TKI prescription within six months of their CML diagnosis, compared to the subsidized, low cost - sharing group.
Sara Mole (UCL) discusses her work on Batten
disease, as well as the universal
challenges encountered when
researching and treating
rare neurological
diseases.