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Orphan Drugs From Regulatory Issues to Clinical Transition

by Mondher Toumi

Part of the Frontiers in Neurotherapeutics Series Series

Orphan Drugs From Regulatory Issues to Clinical Transition Synopsis

While drug discovery for common disorders is somewhat standard and based on the large potential markets, the small market for most orphan diseases means that effective therapies are uncommon. With next generation DNA sequencing, the pace of inherited novel rare disease gene identification has increased markedly. Drug discovery for rare diseases is somewhat hampered by a lack of collaboration between academia and industry separating the necessary resources from skills. This volume discusses important aspects of orphan drug development such as regulatory affairs, public industry partnership, bench to bedside using examples of rare diseases, challenges of clinical trials and future directions.

Book Information

ISBN: 9781498763073
Publication date: 6th August 2019
Author: Mondher Toumi
Publisher: Productivity Press an imprint of Taylor & Francis Inc
Format: Hardback
Pagination: 320 pages
Categories: Medical research, Pharmacology, Biochemistry,

About Mondher Toumi

Dr. Farooq's research focuses on the identification and understanding of the molecular pathways to develop novel therapeutic strategies for neurodegenerative and neuromuscular diseases such as Spinal Muscular Atrophy (SMA), Duchene Muscular Atrophy and Myotonic Dystrophy. He is also a member for the collaborative team of clinicians/scientists at Children's Hospital of Eastern Ontario and other pediatric centers working on initiating a dose escalation trial utilizing FDA approved drug celecoxib for the treatment of human SMA. Dr. Farooq has worked along with Dr. MacKenzie and his team and his research activities are expanding on a wider scale to repurpose drugs for ...

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