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  1. Developing a new orphan/rare disease treatment? Let's talk, our expert scientists can help. Need help with your orphan/rare small molecule program? Contact Quotient Sciences today.

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  1. With more than 1,200 rare disorders, you can explore rare disease reports that include information on symptoms, causes, treatments, clinical trials, and sources of help such as patient advocacy organizations.

    • Achalasia

      Achalasia is a rare disorder that typically affects adults...

    • Aarskog Syndrome

      Noonan syndrome with multiple lentigines (NSML) is a rare...

    • Acoustic Neuroma

      Headaches may also occur in the absence of hydrocephalus and...

    • Abetalipoproteinemia

      Abetalipoproteinemia is a rare inherited disorder affecting...

  2. Feb 1, 2023 · The statutory purpose of this group is “to advise the general assembly regarding research, diagnosis, and treatment efforts related to rare diseases across the state. Key Resources National Organization for Rare Disorders (NORD) Rare Disease Database

  3. A rare disease is a disease that affects a small percentage of the population but there is no single accepted definition. The European Union and United Kingdom define a disease as rare if it affects fewer than 1 in 2,000 people, but there is no universal definition.

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  4. Learn how a disease is defined as rare, why rare disease research is important, what causes rare diseases, and how you can help.

  5. Ohio - National Organization for Rare Disorders. Interested in giving the rare disease community in your state a voice in state government? Learn more about NORD’s work on Rare Disease Advisory Councils (RDACs) and how you can get involved with RDAC activities in your state.

  6. Rare diseases are commonly defined as those with a prevalence of less than 5 in 10,000 people. As of 2021, more than 7000 known rare diseases have been identified.

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  8. Rare Disease: Any disease, disorder, illness, or condition affecting fewer than 200,000 people in the United States is considered rare.¹ 1 in 10 Americans have a rare disease. That is more than 30 million of our loved ones, friends, colleagues and neighbors.² There are more than 10,000 known rare diseases³ and fewer than 5% have an FDA-approved

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