Sentences with phrase «based duchenne»

• Solid Biosciences, a Cambridge, Mass. - based Duchenne muscular dystrophy treatment maker, now says it plans an IPO of 7 million shares priced between $ 18 to $ 19 raising $ 129.5 million.

Not exact matches

• Exonics Therapeutics, Inc, a Boston - based biotechnology company focused on developing SingleCut CRISPR technology to repair mutations causing Duchenne muscular dystrophy and other neuromuscular diseases, raised $ 40 million in Series A funding.

The research, which appears online Aug. 1 in the journal Annals of Neurology, is the first study from a double - blind controlled randomized trial of an exon - skipping agent to provide conclusive proof based on the standard six - minute walk test used to measure muscle function in patients with Duchenne muscular dystrophy (DMD), the most common form of muscular dystrophy in children.

If you took high school biology in the 1990s, you probably learned about the molecular basis for human genetic disorders such as cystic fibrosis (1989), Huntingtons (1993), Duchenne and Becker muscular dystrophy (1987), and a rapidly growing list of single - gene disorders, and the correspondingly rapid growth in clinical diagnostic technology based on DNA sequence information, enabling certain diagnosis, sometimes before the advent of overt symptoms.

Multiplex CRISPR / Cas9 - based genome editing for correction of dystrophin mutations that cause Duchenne muscular dystrophy.