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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

LINE-1 Linked to Premature Aging Conditions

Researchers report in Science Translational Medicine that the accumulation of LINE-1 RNA contributes to premature aging conditions and that symptoms can be improved by targeting them.

Team Presents Cattle Genotype-Tissue Expression Atlas

Using RNA sequences representing thousands of cattle samples, researchers looked at relationships between cattle genotype and tissue expression in Nature Genetics.

Researchers Map Recombination in Khoe-San Population

With whole-genome sequences for dozens of individuals from the Nama population, researchers saw in Genome Biology fine-scale recombination patterns that clustered outside of other populations.

Myotonic Dystrophy Repeat Detected in Family Genome Sequencing Analysis

While sequencing individuals from a multi-generation family, researchers identified a myotonic dystrophy type 2-related short tandem repeat in the European Journal of Human Genetics.