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Development for Rare Disease Treatments
Session Chair(s)
Badri Rengarajan, MD
Life Sciences
Jazz Pharmaceuticals, United States
Planting a Seed: Initiating a Patient Registry in an Ultra-orphan Disease - The Case of Pemphigus and Pemphigoid
Learning Objective : Explain the challenges in establishing a comprehensive patient registry in an ultra-orphan disease area; Identify key criteria for successful implementation of natural history studies in support of development of drugs for orphan diseases; Discuss opportunities to engage with rare disease patient communities and the value to your organization.
Speaker(s)
Planting a Seed: Initiating a Patient Registry in an Ultra-orphan Disease - The Case of Pemphigus and Pemphigoid
Badri Rengarajan, MD
Jazz Pharmaceuticals, United States
Life Sciences
Are Natural History Studies Helpful to Foster Development of New Drugs for Rare Diseases?
Martine Zimmermann, PharmD
Ipsen , France
Senior Vice President, Head of Global Regulatory Affairs, R&D Quality
Reaching a Rare Disease Community Eager for Treatment Options
Brian Loew
Inspire, United States
Chief Executive Officer
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