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Topic: Orphan disease

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	Orphan Products: Hope for People With Rare Diseases
		An orphan disease is defined as a condition that affects fewer than 200,000 people nationwide.
		Many rare diseases or conditions can be difficult to diagnose and manage because in their early stages, symptoms may be absent or masked, misunderstood, or confused with other diseases.
		Congress passed the "Rare Diseases Act of 2002," establishing a role for the ORD at the NIH in encouraging orphan disease research.
	www.fda.gov /fdac/features/2003/603_orphan.html (2485 words)

	WowEssays.com - Orphan Drugs
		Orphan drug also refers to compounds in the public domain for which there can be no patent protection once a company goes through expensive efficacy test and gets FDA approval, so that a competitor could immediately begin producing a generic copy at a fraction of the cost.
		Congressional funding for the orphan drug research grants have hardly been sufficient to fund the number of worthy grant applications submitted by researchers to the FDA (as you will see later on the application is not very easily completed).
		A description of the rare disease or condition for which the drug is being or will be investigated, the proposed indication/s for use of the drug, and the reasons why such therapy is needed.
	www.wowessays.com /dbase/af4/lvw181.shtml (1726 words)

	Orphan Australia - HUNTINGTON'S DISEASE
		Huntington’s Disease (HD), also known by the name of Huntington’s Chorea, is a rare inherited disorder, affecting 1 in 10,000 of the population (0.01%).
		Huntington’s Disease is diagnosed on the basis of the genetic testing for the gene, Huntingtin, found at chromosome 4, or most commonly on the basis of familial history and clinical presentation.
		The course of the disease is progressive but variable; some patients have minimal symptoms, while others deteriorate within 15-30 years with fatality or disability.
	www.orphan.com.au /Tetra_HD.html (528 words)

	Ride For Life \| News Center \| Diseases without clout
		Some cancers belong in the orphan category: One, mesothelioma, is often caused by asbestos contamination of the lungs.
		Orphan disease advocates are speaking out, too, accusing Congress of reneging on its promise.
		Fabry disease usually reveals itself in the teenage years as burning sensations in the hands and feet.
	www.rideforlife.com /news/als_news/diseases_without_clout.html (1533 words)

	Rare Diseases (Site not responding. Last check: 2007-09-17)
		Orphan disease sufferers must often turn to special foundations, such as Ontario's Trillium Foundation, funded through the province's charity casinos, for help with their drug expenses.
		According to Dr. Joe Clarke, an orphan disease specialist at Toronto's Hospital for Sick Children, it remains an "enduring mystery" as to how Genzyme decided on a price for Ceredase since the bulk of the research into the drug's active enzyme was conducted by the publicly funded U.S. National Institutes of Health.
		Garrison said the case is typical of drugs for orphan diseases, which can sit undeveloped on shelves for years unless a pharmaceutical company or government decides to finance the clinical trials that will establish their efficacy.
	www.malattiemetaboliche.it /articoli/troubling_questions.htm (3583 words)

	[No title]
		The approach to orphan drug exclusivity recommended here is that the FDA presume antibodies to the same antigen, of the same immunoglobin class and with the same mechanism of action to be the same drug unless the second antibody is shown to be clinically superior to the first.
		Designation simply requires an FDA determination that the disease indication proposed by the applicant is in fact an orphan disease within the terms of the statute.[33] Designation can be sought at any time during the drug development process and is really without significance unless the drug development process is successful and leads to marketing approval.
		The approach to orphan drug exclusivity recommended here is that the FDA presumes antibodies of the same immunoglobin class and with the same mechanism of action[74] to be the same drug unless the second antibody is shown to be clinically superior to the first.
	www.stlr.org /html/volume5/bohrer.txt (6046 words)

	orphan's challenge
		We are orphans bound by a common thread - a diagnosis of ataxia.
		I challenge you to have the courage to adopt an orphan by the simple and generous act of listening.
		With staggering statistics such as 25 million Americans with rare diseases, chances are you do not have to look far to find an opportunity to have your life enriched by a medical orphan.
	homepage.mac.com /lorrainesutton/Personal19.html (639 words)

	Drug Helps Babies Fight Deadly Botulism - Healthy Kids and Pediatrics - health and medical information produced by ... (Site not responding. Last check: 2007-09-17)
		In fact, the disease is so rare that it has garnered orphan status from the U.S. Food and Drug Administration.
		The FDA passed a law in 1982 allowing manufacturers of drugs that treat orphan diseases to conduct smaller clinical trials, though the drugs must still go through the standard development process and drug makers must prove the safety and efficacy of their drugs.
		From a group of 180 infants believed to have the disease, Arnon and his colleagues were able to enroll 122 infants with suspected (and later lab-confirmed) botulism in their study.
	www.medicinenet.com /script/main/art.asp?articlekey=57670 (828 words)

	Annual Report on the Rare Diseases and Conditions Research Activities of the National Institutes of Health FY 2000 - ... (Site not responding. Last check: 2007-09-17)
		ORLAAM received orphan designation on the basis that it could be used to transfer patients from methadone (and there were fewer than 200,000 receiving methadone), and naltrexone received designation on the basis that there were fewer than 200,000 detoxified addicts at any given time.
		Drug addiction is a disease of the brain, resulting from repeated and prolonged self-administration of such a substance.
		Once the disease is established, however—be it in the brain, the lung, or the heart—the physiological dysfunction must be corrected to restore health.
	rarediseases.info.nih.gov /html/reports/fy2000/nida.html (6479 words)

	Valley fever cure needs federal funding
		Incredible as it seems, one of Arizona's most dangerous and frequent infectious illnesses is an "orphan disease." Because the toll is confined to a few states and not spread across the nation, the federal government considers Valley fever a rare disease.
		The tick-borne disease, which can be debilitating but is almost never fatal, is found in most states but with widely varying severity.
		The Lyme disease rates for the three most badly affected states were 133 per 100,000 in Connecticut, 80 in Rhode Island and 32 in Pennsylvania.
	www.azcentral.com /arizonarepublic/opinions/articles/0115sun1-15.html (643 words)

	Providing Treatments for Rare Diseases: Is the Orphan Drug Act Enough
		Drugs are often “orphaned”, or never are produced and sold on the market, even when a compound is thought to be useful for the treatment of a rare disease.
		In the United States, an “orphan drug” is defined as a drug useful for a condition that is considered to be a rare disease.
		More than 5,000 rare diseases affect millions of people worldwide, yet the patient population for each rare disease is still too small to entice the pharmaceutical industry to offer or manufacture a treatment.
	www.cwru.edu /med/epidbio/mphp439/Orphan_Drug.htm (5883 words)

	(D2OL)™ - Research - Drug Innovation 2000
		For many of these diseases, the newly identified causative gene can be firmly situated within biochemical, signaling or regulatory pathways common to diseases of greater general public health concern such as cancer, heart disease or diabetes.
		When looking for candidates to treat orphan diseases, the first and easiest place to look would be the set of drugs already on the market.
		We believe that the sooner these drugs ‘under development’ are made available to the scientific community championing orphan childhood diseases and diseases common to the third world to test for clinical relevance to these medical conditions, the quicker patients in need can have access to useful and potentially life-saving medicines.
	www.d2ol.com /drug_innovation_2000.html (1216 words)

	FDA approves alglucosidase alfa for Pompe disease (Site not responding. Last check: 2007-09-17)
		Enzyme replacement agent is first ok’d for this orphan disease affecting primarily infants.
		Alglucosidase alfa has not been adequately studied in patients with other forms of Pompe disease, but studies are underway in other patient groups with AAG deficiency.
		Pompe disease manifests as a broad spectrum of clinical symptoms.
	www.pharmacist.com /articles/h_ts_1125.cfm (271 words)

	OOPD Program Overview
		Additional orphan drug amendments were passed by Congress in 1984, 1985 and 1988 The use of the term "orphan", as in "orphan drug", "orphan" disease, etc., does not actually appear in the text of the law which focuses upon definitions of and treatments for "rare diseases and conditions".
		Prior to this revision of the Orphan Drug Act, every sponsor was required to provide financial information regardless of the size of the proposed target patient population.
		A product may still be designated as an orphan by demonstrating that the financial criteria of the law are applicable, regardless of the number of patients affected.
	www.fda.gov /orphan/progovw.htm (1183 words)

	Eurordis - Günther’s disease - Fide Mirón
		Regardless of her terribly mutilated face, despite her hands being so painfully eaten away by the disease, this young Spaniard has an unfailing love of life, holidays in the Canaries and never misses a chance to party with her friends.
		At the age of 29, Fide believes she is the oldest person in Spain to be affected by this disease with the harsh prognosis.
		You should know this: from her participation in the European Conference on Rare Diseases in Luxembourg in June 2005, Fide retains the memory of positive people who are fighting, with an exemplary solidarity.
	www.eurordis.org /article.php3?id_article=841 (526 words)

	Word Spy - orphan drug
		A drug used to treat a rare disease and for which the manufacturer receives special tax credits and marketing rights as an incentive to develop the drug.
		In the decade before it was passed, fewer than 10 orphan drugs came to market; since the act, 193 have been approved, nearly half of them in the last five years.
		Sodium valproate for epilepsy was just such an "orphan drug" and, according to some experts, languished in the bureaucratic pipeline because neither its manufacturer nor the FDA gave it a particularly high priority.
	www.wordspy.com /words/orphandrug.asp (239 words)

	Orphan disease treatment an accidental discovery (Site not responding. Last check: 2007-09-17)
		These are just a few of the symptoms of a potentially fatal condition called Wilson's disease, a recessive genetic disorder that interferes with the body's ability to excrete copper.
		Symptoms are often misdiagnosed as hepatitis, substance abuse or Parkinson's disease, thus delaying treatment and increasing the possibility of permanent organ damage.
		Because Wilson's is considered an "orphan disease"---one that affects fewer than 200,000 people---the recent approval of zinc acetate is especially significant, since most drug companies are unwilling to sponsor orphan drugs for market approval due to the lack of profit potential.
	www.umich.edu /~urecord/9697/May06_97/artcl01.htm (366 words)

	The Rothberg Institute: About TRI: Orphan Disease Proposal
		Many of the 2,000+ compounds currently in clinical development have the potential of addressing orphan diseases, epidemics, and bio-terrorism but they are simply not tested for these purposes.
		Answering to public calls for the pharmaceutical industry to take a more active role in diseases impacting developing nations, Pfizer has recently discovered that their drug Zithromax, used for many years as a treatment for strep throat and other infections, may also be used to treat malaria.
		The goal of the proposal is to have the 2,000+ clinical compounds available so that all orphan disease agencies could work towards curing their respective diseases.
	www.childhooddiseases.org /proposal.html (832 words)

	Institute For Science And Health
		Accordingly, the Institute has adopted the concept of orphan diseases to allow others to gain a better understanding of diseases that are typically under-funded and therefore under-researched.
		There are currently in excess of 6,000 identified orphan diseases, collectively affecting approximately 25 million Americans (or roughly 10% of the current US population).
		The Institute For Science And Health strongly believes that the orphan diseases should not be ignored, but rather vigorously pursued by collaborative research efforts of basic- and clinical-science investigators worldwide.
	www.ifsh.org /Research/Index.asp (481 words)

	Sara's Story
		Dercum's Disease is an Orphan Disease whose cause is not known, which is progressive, incurable and classified as a neurological disease and (possibly) an endocrine deficiency.
		The etiology of the disease is adipose lipoma tumors growing around the peripheral nerves causing intractable pain.
		When you become bed ridden because of this disease, all your muscles will atrophy if you don’t keep up some form of exercise, and swimming is the least painful in my experience.
	members.tripod.com /~Dercums_Data/sara.html (3019 words)

	No
		Initially introduced as orphan diseases by the Orphan Drug Act passed in 1983, these information resources needs to be identified and made readily accessible to the general public, health care professionals, and bio-medical researchers.
		The Genetic and Rare Diseases Information Center and its services are announced in the February 20, 2002 NIH Press Release http://www.nih.gov/news/pr/feb2002/nhgri-20.htm.
		Since the enactment of the Orphan Drug Act, more than 220 new orphan drugs have been approved and marketed in the United States and more than 800 additional drugs are in the research pipeline.
	www.nku.edu /~yannarella/news0209.html (1476 words)

	FCIC - Orphan Products: Hope for People With Rare Diseases
		For example, adrenomyeloneuropathy (AMN), one of a group of genetically determined progressive disorders known as leukodystrophies that affect the brain, spinal cord, and peripheral nerves, is often misdiagnosed as multiple sclerosis (MS), according to the United Leukodystrophy Foundation.
		Since 1983, the ODA has resulted in the development of more than 250 orphan drugs, which now are available to treat a potential patient population of more than 13 million Americans.
		While Stephen C. Groft, Pharm.D., director of the NIH's Office of Rare Diseases, encourages people to use the Internet to find information, he also warns that it is dangerous to rely solely on the computer for medical advice.
	www.pueblo.gsa.gov /cic_text/health/testtube/orphans.htm (1184 words)

	Orphan disease definition - Medical Dictionary definitions of popular medical terms
		Orphan disease: A disease which has not been "adopted" by the pharmaceutical industry because it provides little financial incentive for the private sector to make and market new medications to treat or prevent it.
		A common disease that has been ignored (such as tuberculosis, cholera, typhoid, and malaria) because it is far more prevalent in developing countries than in the developed world.
		The US Orphan Drug Act of 1983 offered tax incentives on clinical trials and 7 years of marketing exclusivity for drugs developed for conditions that occur only rarely in the US.
	www.medterms.com /script/main/art.asp?articlekey=11418 (250 words)

	Genetics of Congenital Central Hypoventilation Syndrome: Lessons from a Seemingly Orphan Disease American Journal of ...
		The initial response to a publication on a pediatric "orphan disease" is not necessarily enthusiasm, especially if the reader is an adult intensivist or pulmonologist.
		Subsequent to the ATS statement we have documented an increase in the number of cases diagnosed, with earlier consideration of the disease.
		CCHS is diagnosed in the absence of primary neuromuscular, lung, or cardiac disease, or of an identifiable brainstem lesion.
	www.findarticles.com /p/articles/mi_qa4085/is_200407/ai_n9452906 (774 words)

	MDAdvice.com - Clinical Trials: Orphan Products
		Blepharospasm is an "orphan" disease--a rare disorder affecting a small population.
		People with diseases such as hemophilia, multiple sclerosis, cystic fibrosis, rare cancers, and scores of other disorders could see little relief in sight.
		Once an orphan drug is approved, exclusivity gives sponsors legal protection against introduction of an identical competing product for seven years.
	www.mdadvice.com /resources/clinical_trials/orphan.htm (1904 words)

	Valley fever aid may be on way
		The University of Arizona plans to seek FDA approval of a promising treatment for valley fever, a lung disease common in the Southwest.
		There are too few people infected with the disease for drug companies to get their investment back.
		Any disease that affects fewer than 200,000 a year is considered by the FDA to be an orphan or rare disease and not considered a major health risk.
	www.azcentral.com /arizonarepublic/local/articles/1217valleyfever.html (555 words)

	Rationing and access to orphan drugs
		Cutting the issue of orphan diseases down to so stark a question — as a paper in last week’s BMJ (2005;331:1016) does — brings the issue into harsh focus.
		The agency arranged reports to be produced on public perspectives of treatments for orphan disease, as well as ethical issues and clinical and cost effectiveness, and sought legal advice.
		The commissioning group’s recommendation on lysosomal storage diseases, endorsed by the boards of all 30 primary care trusts, was not to fund enzyme replacement therapy for Fabry’s disease, MPS1, nor for new patients with Gaucher’s disease.
	www.pharmj.com /Editorial/20051105/news/news_orphandrugs.html (1434 words)

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