Muscular Dystrophy News and Research

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The muscular dystrophies (MD) are a group of more than 30 genetic diseases characterized by progressive weakness and degeneration of the skeletal muscles that control movement. Some forms of MD are seen in infancy or childhood, while others may not appear until middle age or later. The disorders differ in terms of the distribution and extent of muscle weakness (some forms of MD also affect cardiac muscle), age of onset, rate of progression, and pattern of inheritance.

Bringing Maine researchers together to identify solutions to opioid epidemic in Maine

The MDI Biological Laboratory and the University of New England are bringing Maine researchers together to discuss how they can collaborate to identify solutions to the opioid epidemic in Maine.

10 Apr 2019

First-of-its-kind human trial of new brain reading device to help speech and movement

The Stentrode™ is a device that is placed inside a blood vessel of the brain located in an area that controls movement (motor cortex). It is the only investigational technology of its kind that does not require open brain surgery.

8 Apr 2019

Potential drug targets cause of adult-onset muscular dystrophy

People diagnosed with myotonic dystrophy type 1 have difficulty unclenching muscles due to a genetic defect that generates toxic material within their cells.

1 Apr 2019

Gene editing and designer babies experiments face global moratorium

Powerful gene editing tools could soon face a global regulations on their use on human embryos, sperms or eggs.

20 Mar 2019

Call for halt to human gene editing and designer babies experiments

Powerful gene editing tools could soon face a global regulations on their use on human embryos, sperms or eggs.

18 Mar 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

UCL and AIIMS collaborates to increase academic and student exchange

UCL and the All India Institute of Medical Sciences have entered into a new partnership which will enable much closer research collaboration and will explore opportunities to increase academic and student exchange between the institutions.

22 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

Intense exercise, fasting and hormones can enhance waste-protein removal, study shows

The body's ability to adapt to changing conditions and shifting physiologic demands is essential to survival. To do so, each cell must be able to dispose of damaged or unnecessary proteins--a quality-control mechanism critical for cellular performance and for the health of the entire organism.

21 Feb 2019

Newly developed gene therapy helps decelerate aging process

Aging is a leading risk factor for a number of debilitating conditions, including heart disease, cancer and Alzheimer's disease, to name a few. This makes the need for anti-aging therapies all the more urgent. Now, Salk Institute researchers have developed a new gene therapy to help decelerate the aging process.

20 Feb 2019

Unique gene therapy approach paves new way to tackle rare, inherited diseases

Nonsense mutations are single-letter errors in the genetic code that prematurely halt the production of critical proteins. These unfinished proteins are unable to function normally, and nonsense mutations cause 10-15 percent of all inherited genetic diseases, including Duchenne muscular dystrophy, spinal muscular atrophy, cystic fibrosis and polycystic kidney disease.

19 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

Muscular Dystrophy News and Research

Further Reading

Bringing Maine researchers together to identify solutions to opioid epidemic in Maine

First-of-its-kind human trial of new brain reading device to help speech and movement

Potential drug targets cause of adult-onset muscular dystrophy

Gene editing and designer babies experiments face global moratorium

Call for halt to human gene editing and designer babies experiments

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

Johns Hopkins researchers find genetic cause of olfactory neuroblastoma

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

UCL and AIIMS collaborates to increase academic and student exchange

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

Intense exercise, fasting and hormones can enhance waste-protein removal, study shows

Newly developed gene therapy helps decelerate aging process

Unique gene therapy approach paves new way to tackle rare, inherited diseases

Single CRISPR treatment can safely and stably correct genetic disease

Breakthrough study highlights the promise of cell therapies for muscular dystrophy

Study findings hold promise for children with DMD

Researchers unlock key process in human cells that contributes to cancer, neurodegenerative diseases, and aging

New approach to reduce toxic protein production in ALS

Scientists develop new gene therapy that prevents axon destruction in mice

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

Discover how SciY empowers scientists

Why use light scattering to analyze proteins and viral vectors?

Changing the landscape of R&D to build clinical success from the ground up

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