Creating new drugs for disorders like Duchenne Muscular Dystrophy🧐

I am going to talk about another way that the International Space Station Benefits Humanity Back on Earth. It helps researchers to create new drugs for disorders like Duchenne Muscular Dystrophy.

Duchenne muscular dystrophy (DMD) is an incurable genetic disorder that causes progressive muscle weakness and wasting. The disease is caused by a mutation in the DMD gene, which codes for a protein called dystrophin. Dystrophin is essential for muscle function, and its absence leads to the breakdown of muscle fibers. In most cases, muscular dystrophy (MD) runs in families. It usually develops after inheriting a faulty gene from one or both parents.

DMD almost exclusively presents in the XY chromosome combination, people who are most often assigned male at birth. Only rarely does it affect people with the XX chromosome, who tend to be assigned female at birth.

There are more than 30 types of muscular dystrophies, each affecting specific muscle groups and showing symptoms at different ages.

The main sign of Duchenne muscular dystrophy is worsening muscle weakness and loss. Enlarged calf muscles are common, as is pseudohypertrophy, which is caused by a buildup of the fat and connective tissue replacing muscle cells that have died.

Progressive heart enlargement, or cardiomyopathy, is also typical. Most children with Duchenne muscular dystrophy are diagnosed between ages 2 and 11. There is no cure for DMD, nor can muscle damage or loss be reversed. Treatments focus on slowing its progression, managing and alleviating symptoms and complications, and improving a person's quality of life.

A study of the crystal structure of a protein associated with DMD provided hints for compounds that might inhibit it. A professor at the University of Tsukuba in Tsukuba, Japan used these hints to design several promising compounds, including TAS-205. A 2015 study verified the safety of TAS-205 for use in humans. A small clinical trial in human patients was published in 2017. A Phase 3 trial to examine the effectiveness of TAS-205 in situations similar to actual clinical use began in Dec. 2020 and will continue until 2027.

The research team estimates the drug may slow the progression of DMD by half, potentially doubling the lifespan of many patients.

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