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Léon van Wouwe

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Articles

Advocacy in rare disease: Driving technology advances

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By Heidi Rehm, Karin Hoelzer, Simon Alfano, Léon van Wouwe
| Science Magazine Verified
Register NOWRemaining time: 13 DAYS | 17 HRS | 57 MINS Heidi L. Rehm, Ph.D., FACMGSpeaker Karin Hoelzer, D.V.M., Ph.D.Speaker Erika Gebel Berg, Ph.D.Moderator By many measures, this is a golden age of drug discovery, particularly for rare diseases. In 2022, more than half of the novel drugs approved by the U. S. Food and Drug Administration were for patients with rare disease. Many of these treatments represent modalities at the cutting edge of medicine.
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