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Showing articles from gene therapy tag

How A Genetic Counselor Can Help You

Since most rare diseases are caused by changes in your genes, genetic testing is often part of the journey. But understanding your options may be challenging. Partnering with a genetic counselor can help you make the best choice for you and your family. **What is a Genetic Counselor?** A genetic counselor is a h…

The Future of Rare Metabolic Syndromes

Decades of research are yielding new, innovative therapies for rare metabolic syndromes. Learn about the potential for gene therapy, peptide therapeutics, and more. Speakers: James Doyle, PhD, Russell Gotschall and Yael Weiss, MD, MSc. 

Genome Sequencing: Hope, Promises and Limitations

Nearly 80% of all rare diseases have a genetic cause. For many patients, whole-genome sequencing can provide a diagnosis, which can prevent unnecessary additional testing, lead to stronger medical management, and provide a link to a community for support. But not all patients receive a definitive answer. Join experts …

Breakthroughs for Rare Neurological Syndromes

Rare diseases with a neurological component have seen incredible advances in recent years. New therapies have been approved and many more are in the development pipeline. This session will review these advances and consider the promise of future efforts. Speakers: Amit Rakhit, Lale Welsh

Advances in Rare Immunological Diseases

Recent advances in rare immunological diseases have raised the exciting potential of cures for some disorders. Hear from experts about what advances in gene and cell therapies might mean for patients. Speakers: Manish Butte, MD, PhD & Claudia Gonzaga-Jauregui 

Global Genes LIVE!: Harnessing the Power of New Genomic Frontiers

Genomics has accelerated our ability to unlock the mysteries of rare diseases, enabling us to more accurately diagnose and treat patients. Be inspired by leading researchers and advocates as they discuss genomics from the Human Genome Project to novel gene therapies of the future and how they have each leveraged new s…

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