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Rare Diseases

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Can a New Computational Tool Discover and Quantify RNA Molecules More Accurately?

Published on
Mar 23, 2023
in Cornerstone Blog
Snapshot Science: Can a New Tool Read RNA Molecules More Accurately?
Researchers in the Center for Computational and Genomic Medicine developed a new tool to read RNA more accurately.

Enhancing Young Learners’ Knowledge of Rare Diseases

Published on
Feb 28, 2023
in Cornerstone Blog
Enhancing Young Learners’ Knowledge of Rare Diseases
Denise Adams, MD, participated in an FDA-hosted panel discussion about improving young medical professionals’ understanding of rare diseases.

Is Lifelong Immunoglobulin Replacement Therapy Cost-Effective to Treat Agammaglobulinemia?

Published on
Jan 5, 2022
in Cornerstone Blog
Snapshot Science: Is Lifelong Immunoglobulin Replacement Therapy Cost-Effective?
Researchers evaluated whether the standard treatment for a rare disease called agammaglobulinemia is cost-effective in the United States.
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