Rare Disease Advocacy Gone Astray

I’ve been rare for about 14 months now, enough time to start noticing things within the space that aren’t so fabulous. I’m pretty disgusted by the actions of a few people and organizations that are treating rare disease advocacy as some sort of popularity contest.

News flash: If you’re popular with fellow rare disease advocates, you’re doing it wrong!

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Hey FDA, We Need the Orphan Products Board NOW!

In 1983, Congress passed the Orphan Drug Act. The goal was to facilitate the development of drugs and devices to treat rare diseases. Within the legislation was a provision to establish an Orphan Products Board (OPB). The board’s purpose was to orchestrate action between government regulatory bodies, pharmaceutical companies and advocacy groups.

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Achieving an Accurate Diagnosis for Immune Thrombocytopenia

With the prevalence of inaccurate diagnoses issued to rare disease patients, I feel obligated to give an explanation of how my doctors determined I have Immune Thrombocytopenia. While this guide should not be used in place of a healthcare professional’s official diagnosis, you may use this as a guide to request tests so that you can be certain that you have ITP. There are a lot of other diseases and disorders, many of which are autoimmune in nature, that have thrombocytopenia as a symptom. Thrombocytopenia and Immune Thrombocytopenia (ITP) are two entirely different conditions.

*Please note when I discuss my platelet levels, I am referring to them in the 1000 range. So 65 = 65,000*

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