Continuing Advocacy and Expanding Research Efforts: Global Genes 2023 Impact Report

June 7, 2024

To our community members:

For both the rare disease community and Global Genes, 2023 marked a year of significant progress.

There were historic successes with the approval of the first CRISPR Cas 9 gene editing therapy for sickle cell disease, as well as the first gene therapies approved in the United States for hemophilia A, the first gene therapy for Duchenne muscular dystrophy, and the first topical gene therapy for dystrophic epidermolysis bullosa. The pipeline of genetic medicines continues to expand providing hope of not only new therapeutic options for patients, but ones with the potential to provide cures.

For Global Genes, it was a year when we expanded the reach of our efforts to support people with rare diseases, educate the community, and advance research to improve diagnosis and treatments.

Technological innovation coupled with the determination of rare disease patient advocates is accelerating the pace of change to drive research and drug development forward. Global Genes continues to focus on next-generation advocacy to support these efforts, foster collaborations, and gather the data to drive the diagnosis of diseases and the development of therapies to treat them.

This year’s Global Genes Annual Impact Report 2023 reflects the many ways that our organization touched the lives of members of the rare disease community last year through our programs, events, and publications. Thank you for your continued support and participation in these efforts. Together we continue to change what it means to live with a rare disease.

With Hope,

Charlene Son Rigby
CEO, Global Genes  

Download 2023 Impact Report

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