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Orphan Drugs

From Regulatory Issues to Clinical Transition

Mondher Toumi, Ferdinando Squitieri (Herausgeber)

Buch | Hardcover

320 Seiten

2025
Productivity Press (Verlag)
978-1-4987-6307-3 (ISBN)

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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.

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 the treatment of other rare disorders. Dr. MacKenzie and his team are developing a strategic toolbox and preclinical research pathway for inherited disorders and orphan diseases. The lab has identified gene subsets for three disorders – Duchenne Muscular Dystrophy, Myotonic Dystrophy, and Spinal Muscular Atrophy – and the goal is to broaden the research to include other genetic disorders.

Introduction. Policies and Regulatory affairs. Public industry partnership. Advances in NGS and its impact on orphan drug discoveries. Example from rare disorders: SMA- from basic mechanism to phase 3 trial. Duchene Muscular Dystrophy: clinical and therapeutic overview (focusing on current drug pipeline). Other Muscular Dystrophies: Clinical overview. Hereditary Neuropathies. Amyotrophic lateral sclerosis: Current therapeutic pipeline. Familial Dysautonomia. Pamidronate for Rare bone diseases. Repurposing orphan drugs. Orphan Diseases in middle east: Clinical overview. Clinical trials for rare diseases- Lessons learned along with future directions.

Erscheinungsdatum	15.03.2017
Reihe/Serie	Frontiers in Neurotherapeutics Series
Zusatzinfo	8 Illustrations, color; 92 Illustrations, black and white
Verlagsort	Portland
Sprache	englisch
Maße	156 x 234 mm
Themenwelt	Medizin / Pharmazie ► Medizinische Fachgebiete ► Pharmakologie / Pharmakotherapie
	Medizin / Pharmazie ► Pflege
	Medizin / Pharmazie ► Studium ► 1. Studienabschnitt (Vorklinik)
	Naturwissenschaften ► Biologie ► Biochemie
	Naturwissenschaften ► Chemie
	Technik
ISBN-10	1-4987-6307-3 / 1498763073
ISBN-13	978-1-4987-6307-3 / 9781498763073
Zustand	Neuware