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A risdiplam analogue binds to the ESE2 region, which alters the stem-loop structure, TSL1, in the first half of SMN2 exon 7

Systemic functions of bone marrow adipocytes Bone marrow adipocyte-secreted factors in metabolism and muscle function As discussed in Section Bone Marrow Adipose Tissue Functions, BMAT can act as an active secretory organ to produce a variety of factors that could positively or negatively regulate other cellular components, such as SSCs, osteoblasts, osteoclasts, and hematopoietic cells (Figure 19 and Table 1)

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Tambin se estudia su potencial en el tratamiento complementario de trastornos neurodegenerativos como el Alzheimer y la enfermedad de Parkinson