To even be classified in the rare disease category, the disease can only have effected less than 200,000 patients. Defined by NIH, rare diseases are "life-threatening or chronically debilitating diseases which are of such low prevalence that special combined efforts are needed to address them." In total there are about 6,800 rare diseases that affect about 25 million to 30 million Americans in total.
In the world today, there are only about 50,000 cases of patients who are living with a Sarcoma related cancer. Of that population, about 12,000 new cases are diagnosed each year and about 5,000 patients lose their battle each year as well.
A Sarcoma is a rare cancer in adults (1% of all adult cancers), but rather prevalent in children (about 15% of all childhood cancers). It is made up of many “subtypes” because it can arise from a variety of tissue structures (nerves, muscles, joints, bone, fat, blood vessels - these are collectively referred to as the body’s “connective tissues”). Because these tissues are found everywhere on the body, sarcomas can arise anywhere. Thus within each type of the “popular” cancers, there is the occasional surprise for the medical pathologists (e.g., breast sarcoma, stomach sarcoma, lung sarcoma, ovarian sarcoma, etc.). But the most frequent location are the limbs, since this is where the majority of the body’s connective tissue resides. It is often hidden deep in the body, so is often diagnosed when it has gotten too large to enable a hope of cure.
Researchers have made considerable progress in recent years in figuring out ways to diagnose, treat and even prevent a variety of rare diseases. Still, much more remains to be done because there are no treatments for the vast majority of rare diseases.
The Orphan Drug Act of 1983 provides incentives for drug companies to develop treatments for rare diseases. In the 25 years since the Act was signed into federal law, the U.S. Food and Drug Administration (FDA) has approved more than 340 treatments for rare diseases.
Recently, the National Institutes of Health (NIH) launched a new effort, called the Therapeutics for Rare and Neglected Diseases (TRND) program, to create an integrated research pipeline to jump start the development of new treatments for rare and neglected disorders. The NIH Office of Rare Diseases Research (ORDR) handles oversight and governance of TRND. The laboratory work for TRND will be performed in a facility administered by the intramural program of the National Human Genome Research Institute (NHGRI).
To provide patients and their families with timely and reliable information, ORDR and NHGRI have created the online Genetic and Rare Diseases (GARD) Information Center. GARD offers a searchable, frequently updated Web site located at: http://rarediseases.info.nih.gov/GARD. Users can also contact GARD's information specialists directly by e-mail, telephone, FAX, TTY or postal mail.
In addition to GARD, there are many non-profit groups that provide information and support, promote research and advocate for policy issues related to various rare diseases. Among the groups that span a broad range of disorders are the National Organization for Rare Diseases, http://www.rarediseases.org, and the Genetic Alliance, http://www.geneticalliance.org.
Also, Uplifting Athletes, http://www.upliftingathletes.org/, partners with collegiate football teams raising awareness through outreach, research, education and advocacy for rare diseases.
To learn more about specific rare diseases, please check out the National Institutes of Health, Office of Rare Disease Research, http://rarediseases.info.nih.gov/Default.aspx.
Raising Awareness for Rare Diseases .......
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