In the meantime, the focus will be on Amgen's efforts to develop treatments for cancer.
But only 70, 000 people have CF worldwide, too few to lure drug companies to develop treatments.
Ms. Buckley says she appreciates that multiple research groups are trying to develop treatments for Sanfilippo.
"We think that understanding the genes that create risks will help us develop treatments that really are novel, " says Dr. Hyman.
Here are some heroic individuals who, upon discovering they were sick, marshaled resources to develop treatments, changed the way scientists thought and even raised the possibility of cures.
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Thirty years ago, the Orphan Drug Act provided financial incentives to pharmaceutical companies to develop treatments for rare diseases -- diseases that afflict fewer than 200, 000 people.
Their work might help scientists develop treatments for cancer patients.
If companies, hospitals and researchers don't have to cover the full cost of such studies, there might be more efforts to develop treatments for rare diseases, these officials say.
Peers in parliament's upper chamber voted by 212 to 92 on Monday to allow research using stem cells to develop treatments for killer diseases such as leukaemia, Parkinson's and cancer.
Professor Aziz has been working on the brain for decades and is a strong proponent of primate research, having used these animals to develop treatments for neurological diseases such as Parkinson's.
Further into the future, if scientists better understand the neural bases of mental illnesses such as schizophrenia, it could be possible to also develop treatments by targeting groups of neurons with electrical impulses.
Analysts have interpreted that as giving Genzyme's treatment, Fabryzyme, the inside track toward getting a six-year term of exclusivity under orphan drug laws, which is meant to encourage companies to develop treatments for rare diseases.
The project will use supercomputer-based models and simulations to reconstruct a virtual human brain to develop new treatments for neurological conditions.
They added that their results could one day be used to develop new treatments for people who have difficulty forgetting painful events.
Giusti founded an advocacy group, the Multiple Myeloma Research Foundation, that works with companies like Novartis, Celgene, and Merck to develop new treatments.
Merck (nyse: MRK - news - people ) is working to develop obesity treatments that work through the brain, but they are many years away.
Professor Nizetic suggested future research should be directed into basic molecular mechanisms that could one day develop into treatments to children with Down's syndrome in the first few years of life when the brains are "plastic" and rapidly developing.
In a speech at the annual meeting of the American Society of Human Genetics on November 6, Francis Collins, the director of the National Institutes of Health and the man who headed the Human Genome Project, implored his colleagues in genetics to work to develop new treatments for rare diseases.
Patients who stop taking such drugs risk having the virus develop resistance to treatments.
It is an amazing example of how the health care industry can develop personalized cancer treatments.
Microbial genetics is enabling a more precise definition of bacterial, viral and parasitic threats - and how they develop resistance to treatments.
The team hope the findings help to develop new cancer treatments.
He also has investments in such outfits as Euromedic International, which operates diagnostic medical centers throughout Europe, and Broncus Technologies, which is working to develop lung disease treatments.
Her death comes as many of the companies that hoped to use cloning science to develop human medical treatments are struggling for survival, or simply going out of business.
And I am ecstatic that researchers continue to develop wonderful new treatments for more persistent cancers, like breast cancer (a diagnosis my wife received less than two years ago).
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It is also that with so many sufferers being diagnosed too late for surgery, there hasn't historically been as much tissue available to researchers to carry out the kind of genetic research necessary to develop effective targeted treatments.
Early efforts are under way at specialty pharmaceutical firm Allergan and elsewhere to develop anti-inflammatory treatments for the back of the eye.
In an unusual deal, the big drug maker is funding the creation of a biotech company in San Diego called EyeCyte, which will develop stem-cell treatments for eye diseases.
They hope to gain clues to future treatments and develop diagnostic tests that can tell when a person is still "in there" even when he or she cannot communicate.
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