Rare diseases need attention

To the editor:

Happy Rare Disease Day 2017 to my fellow Iowans. I’m sure you’re asking what is Rare Disease Day and how does it affect me?

Rare Disease Day occurs every year on the last day in February. It was created by NORD (National Organization of Rare Diseases) to raise awareness for rare diseases. Diseases are considered “rare,” in the US if the disease infects less than 200,000 people in Uthe .S. There are around 7,000 rare diseases with numerous new ones being discovered everyday. Some rare diseases range from ALCS to GM1 Gangliosodisis to Wolff Parkinson White Syndrome.

Rare diseases affect every shape, size and segment of the population, and are considered by many experts as the epidemic that doesn’t get any press. Statistics suggest 1 out of every 10 of us Americans are affected by rare diseases. That stat equals to over 30 million Americans that are affected. Rare diseases affect more people than cancer and aids combined. It is estimated that 350 million people worldwide are affected. Yet, rare diseases in the United States are underfunded and many cases not funded.

A segment of the population that is particularly hit hard by this epidemic are children. They account for around 50 percent of the people affected. Thirty percent of these children fail to see their fifth birthdays. Rare diseases also account for 35 percent of deaths in children first year of life. Yet, there is 95 percent of these diseases that don’t have one single FDA approved drug treatment. We must find a way to accelerate research and accelerate the need to find cures for these children.

Despite the grim stats, there’s still hope. Since the Orphan Act in 1983, over 300 drugs have been brought on the market to help combat rare diseases. This past year, advocates like Gene Giraffe Project, helped pass the 21st Century Care Act. 21st Century Cures Act enacted $6 billion in funding for research and helped accelerate drugs for FDA approval. Yet, there’s still plenty of work yet to do.

In conclusion, we must start speaking up for those of us who don’t have a voice. We must start fighting for the others who can’t. We must give these children without a chance a chance. We must stand together to fight this epidemic. Frankly, our lives and 30 million Americans affected depend on it.

Doug Passow

President of

Gene Giraffe Project



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