Did you know…

A rare disease is one that affects fewer than 200,000 Americans at any given time.  HIBM is one of those rare disease.

Though the diseases, and many times the symptoms, are uncommon to most doctors, rare diseases – as a whole – represent a large medical challenge.  Combine this with the lack of financial or market incentives to treat or cure the diseases, and you have a serious public health problem.

In 1983 the U. S. Congress enacted the Orphan Drug Act to provide incentives for medical pharmaceutical and product developers to focus on treatments for victims of rare diseases.  Since that time, there have been over 18,000 products studies as possible treatments for rare diseases, and 326 of those have been approved by the FDA.  Most of these were for diseases that had no approved treatment.  Imagine the relief that came to those few patients.

Over the years rare diseases, with the help of NIH, NORD (National Organization for Rare Diseases), Global Genes Project, and the development of non profits championing their support for a rare disease, like ARM, have increased presence in the healthcare landscape and is working hard in giving rare diseases a voice and a chance at treatment development.  More often than not, rare diseases have been labeled as easier and more likely to find treatment than diseases like aids or cancer.  Support a rare disease.  Support HIBM.  Just because you are rare, does not  mean you should not have a voice or a chance at treatment.

  • Approximately 7,000 rare disorders are known to exist and new ones are discovered each year
  • Rare disease affects between 25-30 million people in the United States and approximately 30 million people in the European Union
  • One in 10 Americans is living with a rare disease
  • Children, and young adults, represent the vast majority of those afflicted with rare disease
  • Approximately 80 percent of rare diseases are not acquired; they are inherited. They are caused by mutations or defects in genes
  • In the United States, rare diseases are defined as those affecting 200,000 or fewer people or about 1 per 1,000
  • Rare disease is often referred to as an “orphan” disease
  • Orphan or rare diseases are often not pursued by the pharmaceutical industry because they provide little financial incentive for the private sector to make and market new medications to treat or prevent them and because there are not enough patients to make research cost-effective
  • Research on rare diseases can often lead to advances in our understanding of common diseases such as cancer, heart disease, diabetes, stroke and other major health problems
  • As a whole, rare diseases represent a large medical challenge. Combine this with the lack of financial incentives to treat or cure rare diseases, and a serious public health issue is created
  • The US Orphan Drug Act (ODA) of 1983 has been one of the most successful pieces of health related legislation ever enacted in the United States. Through a system of tax credits, government grants, assistance for clinical research, as well as seven years marketing exclusivity, the Orphan Drug Act has resulted in hundreds of approved orphan medicines, treating over millions of patients worldwide. Similar legislation has been adopted in Japan, Australia and the UK



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