Zebrafish model for an ultra-rare genetic disease reveals two promising drug candidates

Can a small fish help identify possible treatments for an ultra-rare inherited disease found in an Alabama boy? The genetic disease is XMEA, which progressively weakens the muscles and can affect the liver and heart. XMEA stands for X-linked myopathy with excessive autophagy. As of March 2024, only 33 cases had ever been seen worldwide.

This article was originally published on MedicalXpress.com

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