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321 In rare cases, GPI deficiency also affects nonerythroid tissues, causing severe neuromuscular symptoms and granulocyte dysfunction

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This may have contributed to individuals presenting at a relatively late stage in their disease course

Furthermore, small molecule inhibitors of GDF8/Activin (KER-065) and antibodies of its receptor ActRII (Bimagrumab) have shown promising results in promoting weight loss while preserving muscle mass in both animal models and clinical trials (Heymsfield et al