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Rare Community Profiles: The Third Elephant on the Ramp to the Ark: How Carl’s Advocacy Affected His iMCD Journey

Rare Community Profiles: The Third Elephant on the Ramp to the Ark: How Carl’s Advocacy Affected His iMCD Journey

  Rare Community Profiles is a Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their families, advocates, scientists, and more.…

Continue Reading Rare Community Profiles: The Third Elephant on the Ramp to the Ark: How Carl’s Advocacy Affected His iMCD Journey
NXP800 for Cholangiocarcinoma Earns Orphan Drug Designation

NXP800 for Cholangiocarcinoma Earns Orphan Drug Designation

  In the United States, the FDA grants Orphan Drug designation to drugs or biologics that are intended to treat, diagnose, or prevent rare conditions. A rare condition is one…

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The FDA Grants Orphan Drug Designation to AVB-001 for Mesothelioma

The FDA Grants Orphan Drug Designation to AVB-001 for Mesothelioma

  Mesothelioma can sometimes be difficult to treat. The cancer can spread to nearby tissue and nerves, and often isn’t diagnosed until later stages. Admittedly, there are a number of…

Continue Reading The FDA Grants Orphan Drug Designation to AVB-001 for Mesothelioma
Rare Classroom: Stiff-Person Syndrome

Rare Classroom: Stiff-Person Syndrome

Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…

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Implanted “Drug Factories” Successfully Treat Mice with Mesothelioma

Implanted “Drug Factories” Successfully Treat Mice with Mesothelioma

  ScienceAlert recently published an article explaining how scientists at Rice University have been able to eradicate tumors in mice by using a new treatment that takes less than a…

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Rare Classroom: Acanthosis Nigricans

Rare Classroom: Acanthosis Nigricans

Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…

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World Orphan Drug Congress: Rare Disease Trials and Patient Registries

World Orphan Drug Congress: Rare Disease Trials and Patient Registries

On April 28th, the virtual World Orphan Drug Congress USA 2021 was held. The program featured a variety of subjects relevant to the development of orphan drugs and the rare…

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Dermatomyositis: Woman’s Death Highlights Need for More Awareness

Dermatomyositis: Woman’s Death Highlights Need for More Awareness

According to a story from dailymail.co.uk, 59 year old Marcia Ferguson-Roa recently died only a few weeks after unusual symptoms, such as fatigue and ulcers on her head, began to…

Continue Reading Dermatomyositis: Woman’s Death Highlights Need for More Awareness
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Metastatic Breast Cancer: Navigating Grief


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Rethinking What It Means to Live With Acromegaly


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The Let’s Chat CAR T One-on-One Mentor Program: Speaking with Someone Who Understands What You Are Going Through

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