EURORDIS (European Rare Diseases Organisation) organised a Dinner Debate on March 3rd 2009 at the European Parliament in Brussels hosted by MEP Prof Trakatellis, Parliament Rapporteur for the Council Recommendation on the ocassion of Rare Disease Day

CheckOrphan NewsFlash for Tuesday, December 1, 2009: breaking news about rare diseases, orphan diseases, orphan drugs, and neglected diseases. Today’s broadcast highlights topics about: Vanderbilt University Medical Center, colorectal cancer, Ménétrier’s disease, gastrectomy, cetuximab, Erbitux, NHS, National Institute for Health and Clinical Excellence, Innovation Pass, ChemGenex Pharmaceuticals, EMEA, European Medicines Agency, Marketing Authorization Application, omacetaxine, chronic myeloid leukemia, T315I, New Drug Application, Food and Drug Administration, Journal of Neuroscience, Yale School of Medicine, ghrelin, Parkinson’s disease, dopamine neuron, dopamine, lead, amyotrophic lateral sclerosis, Lou Gehrig’s disease, Neurobiology of Disease, Instituto Clemente Estable, University of the Republic, Montevideo, Uruguay, Oregon State University, National Institutes of Health, www.checkorphan.org http

Pheochromocytoma is a rare catecholamine-secreting tumor derived from chromaffin cells. Pheochromocytomas are usually benign (non-cancerous), but can cause dangerously high blood pressure and other symptoms, including pounding headaches, heart palpitations, flushing of the face, nausea, and vomiting. Pheochromocytoma may occur as a single tumor or as multiple growths. It usually develops in the medulla (center or core) of one or both adrenal glands. If the diagnosis of a pheochromocytoma is overlooked, the consequences could be disastrous, even fatal; however, if a pheochromocytoma is found, it is potentially curable.

If you have a pheochromocytoma, your adrenal glands can produce too much of certain hormones, raising your blood pressure and heart rate. Most pheochromocytomas start inside the adrenal gland (the adrenal medulla) where most chromaffin cells are located. There are two adrenal glands, one above each kidney in the back of the upper abdomen. Pheochromocytomas may be inherited, as they are in 10% of patients. Familial cases these tend to occur in younger patients.

Pheochromocytomas often cause the adrenal glands to make too many hormones called catecholamines. Most of the time, a pheochromocytoma is noncancerous (benign), and treatment can return blood pressure to normal. Pheochromocytomas occur most frequently in young to middle-aged adults between the ages of 30 and 60. Pheochromocytomas can be found anywhere in the body, though most occur in the adrenal medulla.

Even a tiny benign pheochromocytoma can make a person very sick. Some people who develop pheochromocytomas have a rare inherited condition, called multiple endocrine neoplasia, that makes them prone to tumors in the thyroid, parathyroid, and adrenal glands. Pheochromocytomas are, fortunately, quite rare and the vast majority of them are entirely benign. Only very rarely is a pheochromocytoma malignant. A pheochromocytoma may be life-threatening if unrecognized or untreated.

Causes of Pheochromocytoma

The common causes and risk factor’s of Pheochromocytoma include the following:

Adrenal gland tumor.

A taxia-telangiectasia.

Dopamine antagonists.

Drugs that inhibit catecholamine reuptake, such as tricyclic antidepressants and cocaine.

Multiple endocrine neoplasia (MEN) syndromes.

Cold medications.

Tumor in the sympathetic nervous system.

Symptoms of Pheochromocytoma

Some sign and symptoms related to Pheochromocytoma are as follows:

Headaches (severe).

Nervousness.

Excess sweating (generalized).

Rapid heart rate.

Abdominal pain.

Hypertensive retinopathy.

Weight loss.

Shaking (tremors) of your hands.

Dizziness.

Feeling of anxiety.

Treatment of Pheochromocytoma

Here is list of the methods for treating Pheochromocytoma:

Stabilization of the person’s vital signs with medication prior to surgery is important, and may require hospitalization.

Surgical tumor removal.

Alpha blockers, also called alpha-adrenergic blocking agents or alpha-adrenergic antagonists, relax certain muscles and help small blood vessels remain open.

Phenoxybenzamine is generally given to stop hormone secretion.

Beta blockers, also known as beta-adrenergic blocking agents, make the heart beat more slowly and with less force.

In the rare instances of pheochromocytomas that are malignant, chemotherapy may be required after surgery.

In 1989, Ft Detrick biological diseases experts were called on by the Virginia Department of Health and Hygiene when a commercial laboratory animal holding facility in Reston, Virginia, experienced an outbreak of an Ebola virus in its primate population. The potentially devastating outbreak was quickly handled, preventing what health officials feared could have had a terrible effect on humans. Author Richard Preston chronicled in The Hot Zone the rapid and effective response by Ft. Detrick scientists and soldiers. This in turn sparked at least one major movie (Outbreak), which based its story on the basic scenario of the Ebola outbreak The US Army established its offensive Biological Warfare program at Fort Detrick in 1943. The purpose of the program was twofold: develop defensive mechanisms against biological attack and develop weapons with which the United States could respond in kind if attacked by an enemy who used biological weapons. The offensive Biological Warfare program remained at Fort Detrick until November 1969, when the United States formally renounced the use of lethal biological agents and weapons, and all other methods of biological warfare and discontinued these offensive programs. From 1943 through 1953, biological warfare research on humans was observational in that it was done after occupational exposure incidents or accidents among workers in the biowarfare facilities. These incidents provided the station hospital the unique opportunity to study the …

The National Organization for Rare Disorders (NORD) provides advocacy, education, and awareness on behalf of all Americans with rare diseases. Most rare diseases still have no treatment, and when there is a treatment, patients often don’t have access to it as a result of reimbursement or insurance issues. Thought leaders from government, patient organizations and industry gathered in Washington DC recently to discuss how to encourage innovative research and how to ensure that patients have access to treatments for rare diseases. Part 3 of 3.

who were previously denied treatment due to preexisting condition restrictions now have life-time comprehensive medical coverage which will cost millions per individual, billions looking at a larger picture?

Do the death panels/bioethics committees tell them no we can’t treat you?

I’m having a little trouble understanding the financial aspects of how the US is going to cover every single US citizens rare or expensive disorder/disease when in the past they had no options due to cost also assuming they can’t deny them coverage based on a preexisting condition. Any ideas?
Mutt, as I understand even if you can afford coverage, you still have the option of using the universal health care given you pay into it and it may be more comprehensive than the private coverage most pay for.

Guillain-Barre syndrome (also known as acute inflammatory or post-infective polyradiculoneuropathy) is a rare but serious disease of the peripheral nervous system. GBS can affect anybody. It can strike at any age and both are equally prone to the disorder. It often follows a minor infection, usually a respiratory (lung) infection or gastrointestinal (gut) infection. It can become life-threatening if the respiratory muscles are affected. GBS is believed to result from an autoimmune response, both humoral and cell mediated, to a recent infection or any of a long list of medical problems. The syndrome may occur at any age, but is most common in people of both between the ages 30 and 50. It is frequently severe and usually exhibits as an ascending paralysis noted by weakness in the legs that spreads to the upper limbs and the face along with complete loss of deep tendon reflexes. Adults are more commonly affected than children. Otherwise, recovery takes several months, but most people recover almost completely. In some cases, GBS can be fatal. The first symptoms of this disorder include varying degrees of weakness or tingling sensations in the legs. In many instances, the weakness and abnormal sensations spread to the arms and upper body. It may occur within days or weeks after a viral infection such as influenza (flu) or diarrhea. It may be triggered by pregnancy or a medical procedure, such as a vaccination or minor surgery, or have no evident reason for developing. Because the cause of GBS is unknown, there’s no way to prevent the disease from occurring.

The most compelling case for a direct relation between the Guillain-Barre Syndrome and preceding infections derives from experience with the enteric pathogen Campylobacter jejuni.Guillain-Barr