NDLI: Implementing a Global Expanded Access Program (EAP) for Infantile-Onset Spinal Muscular Atrophy (Type I): Understanding the Imperative, Impact and Challenges.

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Implementing a Global Expanded Access Program (EAP) for Infantile-Onset Spinal Muscular Atrophy (Type I): Understanding the Imperative, Impact and Challenges.

Content Provider	Europe PMC
Author	Yong, Jonathan Moffett, Megan Lucas, Sam
Copyright Year	2019
Abstract	Nusinersen is the first disease-modifying therapy approved for the treatment of spinal muscular atrophy (SMA), a rare genetic disorder characterized by severe progressive muscular atrophy and weakness. An expanded access program (EAP) provides investigational treatment to patients without other treatment options. An EAP providing nusinersen treatment to individuals with the most severe form of SMA, infantile-onset SMA (consistent with SMA Type I), has enrolled over 800 participants as of September 2018, making it one of the largest in rare disease history. The successes, challenges experienced and opportunities for future consideration during the implementation of the nusinersen EAP are discussed.
Related Links	https://europepmc.org/backend/ptpmcrender.fcgi?accid=PMC6598208&blobtype=pdf
ISSN	22143599
Journal	Journal of Neuromuscular Diseases [J Neuromuscul Dis]
Volume Number	6
PubMed Central reference number	PMC6598208
Issue Number	2
PubMed reference number	31127730
e-ISSN	22143602
DOI	10.3233/jnd-190387
Language	English
Publisher	IOS Press
Publisher Date	2019-01-01
Publisher Place	Nieuwe Hemweg 6B, 1013 BG Amsterdam, The Netherlands
Access Restriction	Open
Rights License	This is an open access article distributed under the terms of the Creative Commons Attribution (CC BY 4.0) License. © 2019 – IOS Press and the authors. All rights reserved
Subject Keyword	Compassionate use spinal muscular atrophy expanded access trial rare disease antisense oligodeoxyribonucleotides drug therapy orphan drug antisense oligonucleotides neuromuscular disease
Content Type	Text
Resource Type	Article
Subject	Neurology Neurology (clinical)

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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