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Showing articles from treatment tag

From Molecules To Medicine: How Are New Drugs & Therapies Developed?

This Global Genes™ Toolkit will provide you with an overview of how new drugs are developed and approved in the U.S. and also what the differences are when developing new drugs for rare diseases. This Toolkit is one in a Global Genes series on drug development,** From Molecules to Medicine**, with others in the series…

From Molecules To Medicine: Clinical Research

Patients with rare diseases often look to new treatments for help. These treatments are often new drugs but may also be devices, vaccines, or biologics such as cell-based treatments or gene therapy. Sometimes, these drugs or biologics are still being tested to learn how well they work and how safe they are. These test…

Gene-Based Diagnosis 101: How to Successfully Navigate the Diagnostic Journey

As scientific innovation in genomic medicine continues to provide more hope for therapeutic options, there is a growing need for patients and families to understand how gene-based testing can contribute to an accurate and timely diagnosis. This series of toolkits, videos, and checklists share insights from rare advoc…

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