Guest blog: Rare diseases – moving forward step by step

In Advocacy by Caroline Hornby

rare disease research by orphan druganaut blog

This guest blog post was kindly provided by Orphan Druganaut Blog.

It is usually not by choice, that families and caregivers of rare disease patients become advocates.  Becoming an advocate is usually influenced by personal circumstances and the unplanned twists and mysteries of life that moves one forward to taking action. It is the desire to help and work tirelessly for a solution to the unknown consequences of living with a rare disease. It is the advocate who is proactive pursuing an understanding of rare diseases, to want to find answers, to want to find cures and treatments.

A recent online article, “Creation of Hayden’s Heroes Inspired by 6-Year-Old with Rare Disease” tells the story of Hayden Boerum and his mother, Melissa Golden, and the creation of “Hayden’s Heroes”. Hayden Boerum is a 6-year old from Maryland, diagnosed at age three with the rare disease, Hemophagocytic  Lymphohistiocytosis (HLH). HLH is a rare disorder of the immune system primarily in young infants and children. This can result in organ damage and tumor formation. It is estimated to occur in 1.2 patients/million children. HLH is one type of Histiocytosis, a group of disorders that occur when there is an over production of white blood cells known as histiocytes. Hayden journeyed through multiple procedures, blood transfusions, chemotherapy, and a year ago underwent a double umbilical cord transplant. Melissa Golden decides to form Hayden’s Heroes, to help other children and their families with Histiocytosis.

Hayden’s Heroes is just one of many similar stories, where a rare disease patient’s family takes on the role of an advocate. This is a story repeated over and over again, resulting in everyday ordinary people taking action and becoming extraordinary. To become a rare disease advocate is to relentlessly raise money and awareness, to organize and communicate with different stakeholders, to move forward step by step, and most of all, to take the time to care.

“Care” is the theme for Rare Disease Day 2014. “Join Together for Better Care” is the slogan for Rare Disease Day 2014. Caring for someone with a rare disease has many sides to it: accessing drugs and other forms of treatment, caring for the caregiver, navigating the healthcare system, and much more. The slogan encourages everyone both inside and outside the rare disease community to unite together for the purpose of receiving better care.

For more information on rare diseases and orphan drugs, check out World Orphan Drug Congress USA. Orphan Druganaut Blog is a media partner of this event.

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