Sentences with phrase «of muscular diseases»

The discovery could have an important impact on the treatment of muscular diseases such as myopathies and muscular dystrophies.

«Discovery of a «conductor» in muscle development could impact on the treatment of muscular diseases.»

«This could therefore have an impact on the treatment of muscular diseases, including myopathies and muscular dystrophies.»

Gut development and cancer, ribozymes and RNA catalysis, and the molecular genetics of muscular diseases will all be served up on the menu shortly.

Not exact matches

The treatment was approved for the devastating rare disease Duchenne muscular dystrophy but created major divisions within the FDA because it simply hadn't demonstrated a whole lot of efficacy, meaning the pricey therapy is essentially being subjected to a real - world clinical test.

The saga of Sarepta Therapeutics» rare disease drug Exondys 51, the first - ever treatment for the degenerative movement disorder Duchenne muscular dystrophy, has taken yet another turn.

Reldesemtiv is the subject of three ongoing Phase 2 clinical trials enrolling patients with spinal muscular atrophy, chronic obstructive pulmonary disease and amyotrophic lateral sclerosis.

You face a higher risk of muscular skeletal disorders, obesity, diabetes, cancer, heart disease and more, even if you work out regularly.

NF is more common than cystic fibrosis, huntingtons disease and forms of muscular dystrophy combined!

Testing on the genetic link for cystic fibrosis, Huntington's disease, Duchenne's muscular dystrophy and other diseases indicates that environmental influences, especially the health and habits of the mother, determine to some extent the form and severity of an offspring's disease.

MDA Summer Camp provides thousands of kids with muscular dystrophy and related muscle - debilitating diseases «the best week of the year.»

Toe walking is sometimes the result of cerebral palsy, muscular dystrophy or another generalized disease of nerve and muscle.

The Commissioner is also directed to determine within 18 months of enactment the eligibility of Alzheimer's Disease, muscular dystrophy, dystonia, post-traumatic stress disorder, and rheumatoid arthritis.

Meanwhile, another measure would increase the number of illnesses that would be applicable to a medical marijuana prescription: PTSD, muscular dystrophy, Alzheimer's disease, traumatic brain injury, wasting syndrome, rheumatoid arthritis and lupus.

His bill would limit its application to epilepsy, intractable skeletal muscular spasticity, traumatic brain injury, glaucoma, cachexia, wasting syndrome, Dravet syndrome, HIV / AIDS, Cancer, ALS, multiple sclerosis, muscular dystrophy, Crohn's disease, and terminal illnesses if a patient has a prognosis of less than 12 months to live.

The first and newest FDA - approved treatment of nusinersen doesn't completely cure the disease, so Sumner's research team wanted to see if using ASO - A in conjunction with nusinersen would further improve muscle form and function in mice with spinal muscular atrophy.

According to studies, approximately one out of every 40 individuals in the United States is a carrier of the gene responsible for spinal muscular atrophy (SMA), a neurodegenerative disease that causes muscles to weaken over time.

Spinal muscular atrophy (SMA) is a devastating hereditary disease that is a leading cause of infant and early childhood mortality.

Whilst there I worked on a research project studying the genetics of inherited deafness and also performed routine genetic screening and prenatal diagnoses for diseases such as Duchenne muscular dystrophy and cystic fibrosis.

The major categories of these devastating wasting diseases include: muscular dystrophy, congenital myopathy and metabolic myopathy.

The new study, called FOR - DMD (Finding the Optimum Regimen of Corticosteroids for Duchenne Muscular Dystrophy), will determine whether daily steroid treatment or an alternative regime is more effective in slowing the disease progression and managing side effects.

These are the muscular dystrophies (among which are Duchenne and Becker); motor neuron diseases (including ALS and SMA); the peripheral nerve disorders (CMT and Friedreich's ataxia); inflammatory myopathies; disorders of the neuromuscular junction; metabolic diseases of muscle as well as other myopathies.

Muscular dystrophies are a group of more than 30 genetic diseases characterized by progressive weakness and degeneration of the skeletal muscles that control movement.

According to recent studies, approximately one out of every 40 individuals in the United States is a carrier of the gene responsible for spinal muscular atrophy (SMA), a neurodegenerative disease that causes muscles to weaken over time.

But in disease conditions like muscular dystrophies, satellite cells can't keep up with repeated cycles of injury and are ultimately exhausted or functionally impaired,» Hindi said.

Baur plans to follow up on the unexpected muscular dystrophy finding, asking if NAD is also depleted in some forms of dystrophy and if restoring NAD might help ameliorate certain features of the disease.

Knowing the sex of a fetus is important when the mother is a carrier of an X-chromosome gene that can cause a disease such as muscular dystrophy.

Researchers at the University of Louisville have discovered a mechanism involved in skeletal muscle repair that may enable clinicians to boost the effectiveness of adult stem cell therapies for diseases such as muscular dystrophy.

Researchers who previously showed that a gene therapy treatment could save the lives of dogs with a deadly disease called myotubular myopathy — a type of muscular dystrophy that affects the skeletal muscles — have found that the therapy is long - lasting.

Meanwhile, the gene - rich X is the most intensely studied of the 23 chromosomes, largely because of its role in rendering men vulnerable to an estimated 300 genetic diseases and disorders associated with those mutations — from color blindness to muscular dystrophy to more than 200 brain disorders.

Inheriting a single X chromosome exposes men to a host of X-linked diseases, such as hemophilia or Duchenne muscular dystrophy, and researchers hope to use the new data to understand more fully the role of genes in other X-linked conditions.

Gene Yeo, a professor of cellular and molecular medicine at UCSD, led the research and showed he could target RNA in living cells, a first step toward treating diseases like muscular dystrophy and neurodegeneration.

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.

«Stem cell gene therapy could be key to treating Duchenne muscular dystrophy: Approach developed at UCLA holds promise for 60 percent of patients with the deadly disease.»

The test developed can be carried out on mothers at risk of X-linked genetic recessive diseases including haemophilia and Duchenne muscular dystrophy and mothers at risk of haemolytic disease of the new - born.

Yin's research also has implications for the treatment of other diseases involving muscle damage, including muscular dystrophy.

The team also showed that they could recover muscle growth and function in mouse models of muscular dystrophy, a disease with a known gene mutation.

And the story of the X has been the story of X-linked recessive diseases, such as color blindness, hemophilia, and Duchenne's muscular dystrophy,» Page adds.

Fresh insights into how our cells control muscle development could aid understanding of muscular dystrophy and other inherited diseases.

Dysfunctional autophagy, on the other hand, is implicated in aging and a range of diseases including cancer, neurodegenerative diseases, muscular disorders, diabetes, and obesity.

Myotonic dystrophy, also known as Steinert disease, is the commonest adult form of muscular dystrophy.

The findings suggest that RIPK1 may be involved in a range of other neurodegenerative diseases marked by axonal damage, including multiple sclerosis, certain forms of spinal muscular atrophy and even Alzheimer's disease.

Duchenne muscular dystrophy, a recessive X-linked form of the disease, affects around 1 in 3,600 boys.

Mitochondrial diseases affect the batteries of the cell and can lead to muscular weakness, blindness, fatal heart failure, learning disability, liver failure, diabetes and can lead to death in early infancy.

In the last four years scientists have cloned and sequenced many genes carrying defects that cause disease, including those responsible for Fragile X linked mental retardation, different forms of muscular dystrophy, and most recently Huntington's disease.

«I'm not sure if we will ever cure Duchenne muscular dystrophy, but I'm very hopeful that someday in the future, we will have new therapies that correct the ability of muscle stem cells to repair the muscles of afflicted patients and turn this devastating, lethal disease into a chronic but manageable condition.»

Now a scientist reports that mice engineered to make extra follistatin, which deactivates myostatin, have four times the muscle of regular mice, suggesting a new target for drugs to fight muscle - wasting diseases such as muscular dystrophy.

Still the study is «an important first step to show that manipulating AMPK - nNOS signaling at least has the potential to help muscle function in muscular dystrophy,» says Michele whose lab at the University of Michigan Cardiovascular Research Center focuses on inherited forms of skeletal and cardiac diseases.

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

«Another intriguing aspect of our study is the molecular link we have established between ALS and spinal muscular atrophy or SMA, which is a pediatric motor neuron disease,» says Edens.