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Doing It Themselves

After a biotech stopped working on a drug to treat Duchenne muscular dystrophy, two families in the US bought the drug to develop it themselves in the hopes of helping family members, reports the Wall Street Journal. Through research foundations, the Secklers and the Wickas paid $500,000 for the drug, called halofuginone, and will pay an additional $500,000. While halofuginone does not fix the underlying genetic cause of Duchenne, the Journal says it has been shown to ease symptoms in mouse models. "As a parent, even worse than having no [treatment] prospects was having a prospect that was going nowhere," says Tracy Seckler. The families hired a former biotech executive to oversee the development of the drug.

However, drug development is fraught with complications that can range from lower-than-expected efficacy to running out of money. "The odds are against you picking a winner in drug development," says Pat Furlong, president of Parent Project Muscular Dystrophy.

The Scan

Fertility Fraud Found

Consumer genetic testing has uncovered cases of fertility fraud that are leading to lawsuits, according to USA Today.

Ties Between Vigorous Exercise, ALS in Genetically At-Risk People

Regular strenuous exercise could contribute to motor neuron disease development among those already at genetic risk, Sky News reports.

Test Warning

The Guardian writes that the US regulators have warned against using a rapid COVID-19 test that is a key part of mass testing in the UK.

Science Papers Examine Feedback Mechanism Affecting Xist, Continuous Health Monitoring for Precision Medicine

In Science this week: analysis of cis confinement of the X-inactive specific transcript, and more.