Sentences with phrase «causing duchenne»

• 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 product that Aronin cites, Sarepta Therapeutics» Exondys 51, is the only cleared treatment to treat the protein deficiency which causes Duchenne in the U.S., and has also enjoyed its fair share of controversy.

Frans Hogervorst and his colleagues at the University of Leiden in Holland say they have overcome this problem by modifying a test which they originally developed to detect the defective genes that cause Duchenne muscular dystrophy.

The genetic mutation that causes Duchenne muscular dystrophy was discovered 30 years ago.

Scientists have developed a CRISPR gene - editing technique that can potentially correct a majority of the 3,000 mutations that cause Duchenne muscular dystrophy (DMD) by making a single cut at strategic points along the patient's DNA, according to a study from UT Southwestern Medical Center.

The researchers then removed the genetic mutation that causes Duchenne using the gene editing technology CRISPR - Cas9.

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

Could a new CRISPR gene - editing technique correct a majority of the 3,000 mutations that cause Duchenne muscular dystrophy?

Not exact matches

And Marathon barely did any actual clinical legwork to get the drug cleared for Duchenne — it relied on 1990 - era clinical trial data before tacking on just enough study material to win an approval that doesn't even address the root cause of the disease.

I say «curious» because Marathon came under intense scrutiny for its initial plans to hike the drug's price to $ 89,000 per year even though it's available for a mere pittance in other countries (and doesn't actually address the root causes of Duchenne, but rather just some of its muscle - wasting symptoms).

The results showed some correction of muscles throughout the body, including in the heart — a major victory because heart failure is often the cause of death for Duchenne patients.

Duchenne muscular dystrophy is caused by problems with the body's ability to produce dystrophin, a long protein chain that binds the interior of a muscle fiber to its surrounding support structure.

Duchenne muscular dystrophy is caused by mutations in a huge gene called dystrophin.

Duchenne mutations cause abnormally low production of the dystrophin protein, which in turn causes muscles to degenerate and become progressively weaker.

They had been working with a worm model of Duchenne muscular dystrophy, a severe form of the disease that strikes young boys and is caused by mutations in the gene that encodes the dystrophin protein.

Duchenne muscular dystrophy is caused by a mutation to one of the longest genes in the body.

This balance can be disrupted in diseases such as Duchenne muscular dystrophy, which is caused by the lack of a muscle - specific protein, dystrophin.

«This is just a first step, but we hope this could lead to a treatment for people with this devastating heart condition, which is a leading cause of death for people with Duchenne muscular dystrophy.»

While years of being on the steroids cause growth suppression, osteoporosis and other bad side effects, boys with Duchenne Muscular Dystrophy walk two to three years longer if they take steroids.

Some muscles, including the heart, were corrected by the therapy, and this is considered a major success because heart failure surfaces as the common cause of death among Duchenne sufferers.

He has been involved in the identification of genes altered to cause muscular dystrophy since his 1986 identification of dystrophin as the causative gene in Duchenne muscular dystrophy.

Erg - Duchenne Palsy is a type of birth injury to the brachial plexus that can be caused by medical malpractice.