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Treatment discovered for deadly childhood disease
12-06-2006 · EurekAlert!Researchers have discovered that a treatment involving enzyme replacement therapy dramatically reduces the risk of death in children with Pompe disease, a rare genetic disorder in which most children die before their first birthday. The disorder causes profound muscle weakness and heart and breathing problems and affects as many as one in 40,000 births. The study is published in the online edition of Neurology, the scientific journal of the American Academy of Neurology.
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Keywords: treatment, discovered, deadly, childhood, disease
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- Research unveils new hope for deadly childhood disease
12-12-2007 · EurekAlert!
Investigators at the University of Rochester have uncovered a promising drug therapy that offers a ray of hope for children with Batten disease -- a rare neurodegenerative disease that strikes seemingly healthy kids, progressively robs them of their abilities to see, reason and move, and ultimately kills them in their young twenties. The study, highlighted in the January edition of Experimental Neurology, explains how investigators improved the motor skills of feeble mice that model the disease.
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- Cancer researchers add spice to research against rare neuromuscular disease
03-20-2007 · EurekAlert!
Scientists have discovered a compound that shows promise against a debilitating neurodegenerative condition known as Kennedy's disease, which is caused by a mutant gene. Currently there is no treatment for the inherited disorder, which resembles a slowly progressive form of Lou Gehrig's disease and affects mainly men.
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- Tumor cell activity may provide clues for treating breast cancer in young women
06-03-2007 · EurekAlert!
When women under 50 develop breast cancer the disease tends to be more aggressive and less responsive to treatment than when it occurs in older women. Researchers at the Duke Institute for Genome Sciences & Policy and the Duke University Comprehensive Cancer Center may have discovered a part of the reason why, and their findings may lead to targeted therapies that can help treat patients more effectively.
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- U of M study: Early treatment can reverse heart damage
08-27-2007 · EurekAlert!
University of Minnesota researchers have discovered that treating people who have early cardiovascular abnormalities, but show no symptoms of cardiovascular disease, can slow progression and even reverse damage to the heart and blood vessels.
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- New cataract gene discovered
02-14-2008 · EurekAlert!
Researchers at ETH Zurich and the University Zurich have identified the chromosomal location and exact molecular defect in the coding region of the gene responsible for a childhood cataract. Until now, no human disease could be associated with this gene. The researchers' results will be published in the American Journal of Human Genetics.
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- Infectious disease researchers develop basis for experimental melanoma treatment
12-07-2006 · EurekAlert!
While investigating a fungus known to cause an infection in people with AIDS, two grantees of the National Institute of Allergy and Infectious Diseases, part of the National Institutes of Health, unexpectedly discovered a potential strategy for treating metastatic melanoma, one of the deadliest forms of skin cancer. The treatment approach, which involves combining an antibody with radiation, has since been further developed and is expected to enter early-stage human clinical studies in 2007.
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- Mayo discovers link between Huntington's and abnormal cholesterol levels in brain
12-01-2006 · EurekAlert!
Mayo Clinic researchers have discovered a protein interaction that may explain how the deadly Huntington's disease affects the brain. The findings, published in and featured on the cover of the current issue of Human Molecular Genetics, show how the mutated Huntington's protein interacts with another protein to cause dramatic accumulation of cholesterol in the brain.
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- Jefferson scientists find protein may be key in developing deadly form of pancreatic cancer
10-11-2007 · EurekAlert!
A tumor-blocking protein previously implicated in prostate and breast cancer development may also be behind the most aggressive type of pancreatic cancer. Researchers have discovered that the protein pp32 -- which normally applies the brakes on a cancer-causing gene -- is missing in an aggressive form of pancreatic cancer. Though the work is preliminary, the scientists say, the absent protein could eventually become a marker for the disease and a potential drug target.
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- Chemical chaperone could open door to treatment of neurological disorder
02-05-2008 · EurekAlert!
An unexpected finding turned out to be a clue leading researchers at Washington University School of Medicine in St. Louis to propose a new treatment approach for Niemann-Pick disease, a rare, deadly neurodegenerative disorder. They believe the approach also could be useful for more common diseases -- such as cystic fibrosis -- that stem from a similar type of defect.
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- Rice breakthrough could prevent multiple fibrotic diseases
01-18-2007 · EurekAlert!
A scientific breakthrough at Rice University may lead to the first treatment that prevents the build-up of deadly scar tissue in a broad class of diseases that account for an estimated 45 percent of U.S. deaths each year. Fibrotic diseases include cardiac and pulmonary fibrosis, atherosclerosis, asthma, cirrhosis and scleroderma. Rice researchers have discovered a naturally occurring blood protein that's proven effective at preventing fibrotic scarring in lab animals.
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