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Release date- 26082019 - NEWPORT BEACH - Receiving a positive diagnosis for Duchenne muscular dystrophy is devastating for new parents.
6, 2019-- Vertex Pharmaceuticals Incorporated (NASDAQ: VRTX) today announced that the company is enhancing its gene editing capabilities to develop novel therapies for Duchenne Muscular Dystrophy (DMD) and Myotonic Dystrophy Type 1 (DM1) by expanding its collaboration with CRISPR Therapeutics and acquiring Exonics Therapeutics.
Primarily affecting males, Duchenne muscular dystrophy (Duchenne) is a rare and fatal genetic disorder that results in progressive muscle weakness from early childhood and leads to premature death in the mid-twenties due to heart and respiratory failure.
Faye said: "My sister and I and our husbands are going to take part in the Duchenne Dash.
Felix is the four-year-old grandson of Barrie and Denise Wakeling who was diagnosed with Duchenne Muscular Dystrophy last year.
They restored muscle stem cell function that is impaired in Duchenne muscular dystrophy, resulting in efficient regeneration of the muscle and preventing the progressive loss of muscle strength characteristic of the disease.
The youngsters donned orange clothing to show their support for Somhairle, raising PS400 for research into Duchenne Muscular Dystrophy.
The event is being organised by Catriona and Mike Marshall, who have been so impressed with the rapid progress being made by Duchenne UK, an agile young charity, that they want to help them on their mission to end this terrible disease.
Using a method called the visual rivalry, the researchers showed study participants computer-generated avatars, one with and one without the Duchenne marker, to study which expressions our brains perceive as more important.
A previous clinical trial, the HOPE-Duchenne trial, found a single intracoronary dose of CAP-1002 was generally safe, well tolerated and demonstrated significant and sustained signals of improvement in cardiac and skeletal muscle function in boys and young men in advanced stages of Duchenne muscular dystrophy.
Muscular dystrophies are clinically divided into six types as Duchenne (DMD), Becker (BMD), Limb-girdle (LGMD), Congenital (CMD), Facio-Scapulo-Humeral Muscular dystrophy (FSHMD), distal myopathies and myotonic dystrophy (MD).2

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