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    Grifols Starts Phase 3 Trial for a New, More Convenient Alpha-1 Treatment
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    Grifols Starts Phase 3 Trial for a New, More Convenient Alpha-1 Treatment

    What Happened? The healthcare company Grifols announced in a recent press release that it has enrolled and dosed the very first patient in a new Phase 3 clinical trial called…

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    Diagnosing the Undiagnosed in Rare Disease: Worldwide, Multi-Institution, and Multi-disciplinary Work Underway

    Recently at the World Orphan Drug Conference in Boston, Patient Worthy had the opportunity to meet with Dave Pearce PhD, Chair of International Rare Disease Research Consortium, (IRDiRC) Professor of…

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    Heart Attacks and Your Brain

    1. A Physical Smoking Gun in the Heart-Brain Connection When people experience cognitive problems, mood drops, or chronic anxiety after surviving a heart attack, the medical community traditionally viewed it…

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    Insights into Gazyva as a Treatment for Lupus/SLE

    A recent article from Inside Precision Medicine states: For decades, systemic lupus erythematosus (SLE) has resisted tidy solutions, a shapeshifting autoimmune disease that crosses into different organ systems, flares without…

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    Draft Guidance from FDA Introduces Important Changes to Drug Development for Rare Diseases

    On February 23, 2026, FDA issued a Draft Guidance document, called “Considerations for the use of the Plausible Mechanism Framework to Develop Individualized Therapies that Target Specific Genetic Conditions with Known Biological…

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