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Showing articles from Drug development tag

What is Druggable New Therapeutic Targets in Rare Disease

Only a fraction of the more than 7,000 rare diseases identified have approved therapies available. Advances in DNA sequencing have led to the identification of the genetic cause of several rare diseases, which can potentially lead to a better understanding of the disease and development of targeted therapies. Hear fro…

The Value of Rare Disease Therapies: Patient Perspectives Needed

There is a lot of public debate surrounding the value of rare disease therapies. How do we ensure that the perspectives of patients, families, and advocates are included in these discussions and assessments? Advocates and economic experts will discuss new thoughts on the value of therapies and how advocates can get in…

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