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In the context of rare diseases, the distinct characteristics of patient population introduce additional hurdles compared to trials for more common diseases (Table 1)

Among the 36 sphingolipids, one species was significantly upregulated, while 11 species were downregulated (Figure 4C)

Finally, raising antibodies directly against GPCRs is a task that has proven to be quite difficult (Fredholm et al., 2007), although tagging alternatives are available

This results in diminished proliferation, impaired differentiation capacity, and elevated secretion of pro-inflammatory cytokines, all of which adversely impact therapeutic efficacy

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Ramadani and Abulreesh, 2010