Dystrophin News and Research

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Dystrophin is a rod-shaped cytoplasmic protein, and a vital part of a protein complex that connects the cytoskeleton of a muscle fiber to the surrounding extracellular matrix through the cell membrane.

Experimental treatment shows early promise to help nearly half of DMD patients

A new therapeutic being tested by University of Alberta researchers is showing early promise as a more effective treatment that could help nearly half of patients with Duchenne muscular dystrophy.

25 Sep 2019

Cheaper drug equally effective in preserving cardiovascular function in boys with DMD

A new clinical trial conducted at The Ohio State University Wexner Medical Center found a cost-effective generic medication works just as well as a more expensive drug in preserving cardiovascular function in boys with Duchenne muscular dystrophy (DMD).

24 Sep 2019

Study: Gene editing may correct genetic mutation responsible for Duchenne muscular dystrophy

Duchenne muscular dystrophy (DMD) is a rare but devastating genetic disorder that causes muscle loss and physical impairments.

18 Sep 2019

New drug provides hope for patients with Duchenne muscular dystrophy

The results from three clinical trials have shown that a new drug can successfully delay the progression of Duchenne muscular dystrophy.

10 Jul 2019

New discovery could improve therapies for Duchenne muscular dystrophy

A new multi-institution study spearheaded by researchers at Florida State University and the University of California, Los Angeles suggests a tiny protein could play a major role in combating heart failure related to Duchenne muscular dystrophy (DMD), the most common lethal genetic disorder among children.

16 Jun 2019

Novo Biosciences achieves major milestones in its new drug to treat heart disease and DMD

Novo Biosciences Inc., has achieved several major milestones in its mission of bringing its breakthrough drug candidate, trodusquemine, to market as a potential regenerative medicine treatment for heart disease and Duchenne muscular dystrophy.

15 Apr 2019

Researchers develop new method to boost efficiency of CRISPR gene editing in DMD

Scientists have developed a method to boost the efficiency of CRISPR gene editing in Duchenne muscular dystrophy (DMD), according to a study that could have implications for optimizing gene therapies for other diseases.

7 Mar 2019

Johns Hopkins researchers find genetic cause of olfactory neuroblastoma

By sequencing the entire genomes of tumor cells from six people with a rare cancer of the nose and sinus cavity, Johns Hopkins researchers report they unexpectedly found the same genetic change¾one in a gene involved in muscle formation¾in five of the tumors.

28 Feb 2019

Clinical study shows tolerability and safety of investigational drug in boys with DMD

Patients with Duchenne muscular dystrophy have few treatment options. Medications currently available or in development either target only a subset of DMD patients with a particular genetic mutation or cause significant side effects.

23 Feb 2019

Phase 1 data reinforce safety profile of new drug for treating Duchenne muscular dystrophy

Patients with Duchenne muscular dystrophy (DMD) have few treatment options. Medications currently available or in development either target only a subset of DMD patients with a particular genetic mutation or cause significant side effects.

21 Feb 2019

Single CRISPR treatment can safely and stably correct genetic disease

Researchers at Duke University have shown that a single systemic treatment using CRISPR genome editing technology can safely and stably correct a genetic disease -- Duchenne muscular dystrophy (DMD) -- for more than a year in mice, despite observed immune responses and alternative gene editing outcomes.

18 Feb 2019

Study findings hold promise for children with DMD

Prednisone, the current standard of care used to treat kids with Duchenne muscular dystrophy (DMD), reduces chronic inflammation but has harsh side effects.

11 Feb 2019

Researchers identify and overcome barrier in CRISPR gene editing to treat muscular dystrophy

The gene editing technique known as CRISPR is a revolutionary approach to treating inherited diseases. However, the tool has yet to be used to effectively treat long-term, chronic conditions.

9 Jan 2019

Urine exRNA may be source of biomarkers for muscular dystrophy

Massachusetts General Hospital researchers have found that extracellular RNA in urine may be a source of biomarkers for the two most common forms of muscular dystrophy, noninvasively providing information about whether therapeutic drugs are having the desired effects on a molecular level.

26 Sep 2018

Stanford researchers show how to prevent immune response to gene therapy in mouse model

Stanford University School of Medicine researchers have demonstrated that gene therapy can be effective without causing a dangerous side effect common to all gene therapy: an autoimmune reaction to the normal protein, which the patient's immune system is encountering for the first time.

4 Sep 2018

Muscle dystrophy treatment in dogs with Crispr gene editing

Researchers in the United States have shown that genetically caused muscular dystrophy in dogs could be corrected using genetic editing tools. Muscular dystrophy is one of the most common fatal genetic conditions seen in children and is also seen in dogs

31 Aug 2018

Pfizer terminates clinical studies evaluating domagrozumab for treatment of Duchenne muscular dystrophy

Pfizer Inc. announced today that it is terminating two ongoing clinical studies evaluating domagrozumab (PF-06252616) for the treatment of Duchenne muscular dystrophy (DMD): a Phase 2 safety and efficacy study (B5161002) and an open-label extension study (B5161004).

30 Aug 2018

New project to combat DMD-related fibrosis receives major funding boost

Duchenne muscular dystrophy is one of the most common congenital diseases in the world, affecting one in 3,500 Canadian males. DMD is caused by mutations in the dystrophin gene that results in progressive muscle degeneration and there are currently no effective treatments for DMD.

17 Aug 2018

New cell-based technologies could help improve understanding of muscle-wasting disease

Researchers from Queen Mary University of London have developed new cell-based technologies which could help improve understanding of the muscle-wasting disease Duchenne muscular dystrophy (DMD) and test potential drugs for the disease.

6 Jun 2018

Research establishes use of chimeric cells as potential therapy for Duchenne muscular dystrophy

Cells made by fusing a normal human muscle cell with a muscle cell from a person with Duchenne muscular dystrophy --a rare but fatal form of muscular dystrophy -- were able to significantly improve muscle function when implanted into the muscles of a mouse model of the disease.

16 Mar 2018