Lorenzo had Adrenoleukodystrophy (ALD), a genetic disease that progressively destroys the brains of young boys.
Six month old Seth Petreikis was born with an extremely rare genetic disease called DiGeorge Syndrome.
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There is a rare genetic disease in which people do not have functioning SGLT2.
Thursday, Isis announced trials that could get mipomersen approved in the rare genetic disease familial hypercholesterolemia (FH).
The Neuro Foundation says one baby is born each day in the UK with this genetic disease.
But both have a genetic disease, and, were they to have a child together, that child would suffer terribly.
But, in the Cell study, Mitalipov and colleagues began with skin cells from an 8-month-old baby that had a genetic disease.
For one thing, mipomersen will at first be aimed squarely at the kind of market Genzyme understands best: an inherited genetic disease.
Retinitis pigmentosa is a genetic disease that causes people to slowly lose their eyesight as time goes on and eventually become blind.
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Unlike other cholesterol medicines, Isis' experimental mipomersen will be targeted at patients whose high cholesterol is caused by a relatively rare genetic disease.
The ENHANCE study was conducted in 720 patients with heterozygous familial hypercholesterolemia, a genetic disease that can boost untreated cholesterol levels above 300.
The mice had a genetic disease which affected microscopic hairs in their body - called cilia - which can detect chemicals in the air.
The vote was close, with 9 for approval and 6 against, and only for treatment in a rare genetic disease called homozygous familial hypercholesterolemia (HoFH).
Last month, the European Union approved Glybera for treatment of a rare genetic disease, making it the first gene-therapy medicine approved in the Western world.
Rowley's discoveries of chromosome abnormalities in leukemia secured a common agreement among scientists, physicians and the general public that cancer is, in fact, a genetic disease.
In the artery study, doctors gave 720 patients with a genetic disease that causes high cholesterol either the Vytorin combination pill or just the Zocor component.
At most, the study suggests that people in high risk groups who know they are likely to get a genetic disease can handle negative health information.
The U.S. Food and Drug Administration yesterday approved BioMarin's (nasdaq: BMRN - news - people ) first drug, a treatment for a rare genetic disease.
The passage of the Orphan Drug Act in 1983 made it financially worthwhile for drug companies to chase treatments for disorders like multiple sclerosis, rare cancers, even rare genetic disease.
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This is not a medicine that is likely to be used in patients with garden variety high cholesterol, or even those with less serious versions of the genetic disease FH.
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Aryplase works against MPS VI, another rare genetic disease.
The most recent new cholesterol drugs, Juxtapid from Aegerion Pharmaceuticals and Kynamro from Isis Pharmaceuticals, have focused on patients with a severe genetic disease that causes their cholesterol to be very high.
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The 700 patients in the ENHANCE trial, who all had a genetic disease causing their levels of bad cholesterol to spike without treatment, were all on the top dose of the cholesterol-lowering drug Zocor.
But the nearest-term potential for the drug will be in patients who have a rare genetic disease in which both copies of a gene that controls getting cholesterol out of the body are defective.
Another possibility is that the study was hobbled because many patients in it had a genetic disease that leads to an extremely high cholesterol level and had been treated for a long time on cholesterol drugs.
The other cases do not show the sort of transmission pattern that normally gives away a genetic disease yet there is a bigger chance than normal that someone will suffer from Alzheimer's if relatives on his mother's side have also suffered.
Last night, the Food and Drug Administration announced the approval of Kynamro, a new drug to lower cholesterol in patients with homozygous familial hypercholesterolemia (HoFH), a rare genetic disease that causes sky-high cholesterol levels and heart attacks at a young age.
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See, for instance, this case I wrote about a year ago, or this one, in which sequencing the part of the exome that was on the X chromosome allowed scientists to figure out the cause of a genetic disease that was killing young boys in a Utah family.
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It is a particular gamble if the child is conceived partly to provide a bone marrow graft for a brother or sister with a nasty genetic disease: not only might the new baby turn out be a poor match for transplantation, but might also have inherited the same disorder as his or her sibling.
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