NDLI: Drug Development for Rare Mitochondrial Disorders

Content Provider	Springer Nature Link
Author	Hurko, Orest
Copyright Year	2013
Abstract	Currently, all treatment of mitochondrial disorders is performed with dietary supplements or by off-label use of drugs approved for other indications. The present challenge is translation of our collective knowledge of the molecular details underlying the pathophysiology of mitochondrial disorders into safe and effective therapies that are approved by the regulatory authorities. Molecular details permit precise diagnoses, but homogeneity is gained at the expense of limiting numbers of subjects for clinical trials and of small markets from which to recoup the considerable expense of drug discovery and development. The Food and Drug Administration recognizes that trial designs suitable for common diseases are often not feasible for rare disorders. They have developed a number of programs to facilitate development of novel therapies for such rare diseases, without compromise of regulatory standards. With advances in technology, including the use of biomarkers, replacement therapies and sophisticated trial designs, both biotechnology firms and, increasingly, large integrated pharmaceutical companies, are taking advantage of the opportunities in rare disorders. Precise molecular delineation of pathophysiology and of responsive patients has led to success rates with rare diseases that are significantly greater than those for common disorders. It appears likely, but not yet proven, that this may now be the case for rare mitochondrial disorders as well.
Starting Page	286
Ending Page	306
Page Count	21
File Format	PDF
ISSN	19337213
Journal	NeuroRX
Volume Number	10
Issue Number	2
e-ISSN	18787479
Language	English
Publisher	Springer-Verlag
Publisher Date	2013-02-21
Publisher Institution	American Society for Experimental NeuroTherapeutics
Publisher Place	New York
Access Restriction	One Nation One Subscription (ONOS)
Subject Keyword	Drugs Food and drug administration Clinical trials Rare diseases Biomarkers Mitochondrial Neurosciences Neurology Neurosurgery Neurobiology
Content Type	Text
Resource Type	Article
Subject	Neurology (clinical) Pharmacology Pharmacology (medical)

Sl.	Authority	Responsibilities	Communication Details
1	Ministry of Education (GoI), Department of Higher Education	Sanctioning Authority	https://www.education.gov.in/ict-initiatives
2	Indian Institute of Technology Kharagpur	Host Institute of the Project: The host institute of the project is responsible for providing infrastructure support and hosting the project	https://www.iitkgp.ac.in
3	National Digital Library of India Office, Indian Institute of Technology Kharagpur	The administrative and infrastructural headquarters of the project	Dr. B. Sutradhar bsutra@ndl.gov.in
4	Project PI / Joint PI	Principal Investigator and Joint Principal Investigators of the project	Dr. B. Sutradhar bsutra@ndl.gov.in Prof. Saswat Chakrabarti will be added soon
5	Website/Portal (Helpdesk)	Queries regarding NDLI and its services	support@ndl.gov.in
6	Contents and Copyright Issues	Queries related to content curation and copyright issues	content@ndl.gov.in
7	National Digital Library of India Club (NDLI Club)	Queries related to NDLI Club formation, support, user awareness program, seminar/symposium, collaboration, social media, promotion, and outreach	clubsupport@ndl.gov.in
8	Digital Preservation Centre (DPC)	Assistance with digitizing and archiving copyright-free printed books	dpc@ndl.gov.in
9	IDR Setup or Support	Queries related to establishment and support of Institutional Digital Repository (IDR) and IDR workshops	idr@ndl.gov.in

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