DMD is a well-characterized X-linked disorder caused by mutations ... Regarding the MD group, these patients did not receive MLPA, single-gene sequencing, or gene panel analysis prior to WES. They ...

Technological advances have reduced genome sequencing costs to $600 ... (AOC 1044), a treatment for people living with Duchenne muscular dystrophy amenable to exon 44 (DMD44).

These work by binding to specific mRNA sequences in order to modify gene expression. By correcting faulty ... with AIDS – was cleared by the FDA in 1998. For people with Duchenne muscular dystrophy, ...

Duchenne muscular dystrophy (DMD) is a rare hereditary disease that is associated with progressive muscle wasting. The ...

Gene therapies have set a high bar for outcomes in pharma by delivering curative treatments for difficult diseases in a one-and-done punch. But for Duchenne muscular dystrophy, the results of ...

Researchers at the University of Toronto have invented a new method that uses DNA sequencing to measure metabolites. This ...

Researchers at the University of Toronto have invented a new method that uses DNA sequencing to measure metabolites. This ...

The research is published in the journal Nature. The study comprehensively analyzed "cis-regulatory elements" (CREs), which are the DNA sequences that regulate gene transcription. This work sheds ...

Duchenne muscular dystrophy (DMD) is a rare genetic disorder that is characterised by progressive muscle degeneration and weakness, and is caused by mutations in the dystrophin gene. Currently ...

The phase 3 EMBARK trial has been evaluating the gene therapy in ambulatory boys aged four to seven years with a confirmed mutation in the DMD gene. Estimated to affect one in every 5,000 male births ...