TABLE OF CONTENTS

Volume 24, Issue 9 (September 2017)

Spinraza And Advanced Therapies: A Stakeholder Special

The approval of Spinraza (nusinersen) for the treatment of the severe rare disease Spinal muscular atrophy (SMA) is a breakthrough in the Advanced Therapies field and highlights the importance of involving all stakeholders for the successful development of therapeutics. The mechanism of action of Spinraza is based on a thorough understanding of the molecular basis of SMA and shows how genetic therapies can be rationally designed when significant knowledge about the disease has been gathered. The availability of Spinraza, a treatment with broad label, impacts a large number of people, from babies and infants through to adults. It also has implications for the possible introduction of newborn screening and showcases the difficulty in providing affordable access to advanced treatments. The Spinraza and Advanced Therapies: a stakeholder special issue of Gene Therapy covers a variety of perspectives and includes comments and review papers from SMA research funders, people affected, experts in the advanced therapies field, SMA scientists and clinicians, regulators, ethics and pricing/IP experts, and community stakeholders. The issue also covers genomics projects for personalised medicine, the importance of international collaboration in rare disease research, showcases on-going research in Asia, South America and Africa, and provides academic and corporate perspectives on the delivery of advanced therapies. It also includes a compilation of online resources of relevance. This content shows the global, inclusive and accessible way in which we want to develop the journal.

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Gene Therapy, more than ever—a new vision for the journal  

R J Yáñez-Muñoz

Gene Ther 24: 493-494; doi:10.1038/gt.2017.60

Gene therapy—from small beginnings to where we are now  

J C Glorioso and N Lemoine

Gene Ther 24: 495-496; doi:10.1038/gt.2017.23

Spinraza—a rare disease success story  

V Prakash

Gene Ther 24: 497; doi:10.1038/gt.2017.59

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Cure SMA and our patient community celebrate the first approved drug for SMA  

J Glascock, M Lenz, K Hobby and J Jarecki

Gene Ther 24: 498-500; advance online publication, June 1, 2017; doi:10.1038/gt.2017.39

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Spinraza—the patient perspective  

S Madipalli

Gene Ther 24: 501-502; advance online publication, September 7, 2017; doi:10.1038/gt.2017.56

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Gene therapy for spinomuscular atrophy: a biomedical advance, a missed opportunity for more equitable drug pricing  

T Friedmann

Gene Ther 24: 503-505; advance online publication, June 22, 2017; doi:10.1038/gt.2017.48

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Developmental regulation of SMN expression: pathophysiological implications and perspectives for therapy development in spinal muscular atrophy  

S Jablonka and M Sendtner

Gene Ther 24: 506-513; advance online publication, June 29, 2017; doi:10.1038/gt.2017.46

Therapeutic approaches for spinal muscular atrophy (SMA)  

M Scoto, R S Finkel, E Mercuri and F Muntoni

Gene Ther 24: 514-519; advance online publication, June 29, 2017; doi:10.1038/gt.2017.45

How the discovery of ISS-N1 led to the first medical therapy for spinal muscular atrophy  

N N Singh, M D Howell, E J Androphy and R N Singh

Gene Ther 24: 520-526; advance online publication, May 25, 2017; doi:10.1038/gt.2017.34

Development of gene therapies—lessons from nusinersen  

L Xu, I Irony, W W Bryan and B Dunn

Gene Ther 24: 527-528; advance online publication, September 7, 2017; doi:10.1038/gt.2017.64

The clinical landscape for SMA in a new therapeutic era Open  

K Talbot and E F Tizzano

Gene Ther 24: 529-533; advance online publication, July 20, 2017; doi:10.1038/gt.2017.52

New treatments for serious conditions: ethical implications  

N M P King and C E Bishop

Gene Ther 24: 534-538; advance online publication, May 11, 2017; doi:10.1038/gt.2017.32

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Market access of Spinraza (Nusinersen) for spinal muscular atrophy: intellectual property rights, pricing, value and coverage considerations  

S Simoens and I Huys

Gene Ther 24: 539-541; advance online publication, September 7, 2017; doi:10.1038/gt.2017.79

Will rising prices and budget constraints prevent patients from accessing novel gene therapies?  

A Kent and J Spink

Gene Ther 24: 542-543; advance online publication, September 7, 2017; doi:10.1038/gt.2017.66

The SMA Trust: the role of a disease-focused research charity in developing treatments for SMA  

V Christie-Brown, J Mitchell and K Talbot

Gene Ther 24: 544-546; advance online publication, June 22, 2017; doi:10.1038/gt.2017.47

Building the patient community  

F Raffai and O Timmis

Gene Ther 24: 547-550; advance online publication, May 11, 2017; doi:10.1038/gt.2017.33

Human genomics projects and precision medicine  

F Carrasco-Ramiro, R Peiró-Pastor and B Aguado

Gene Ther 24: 551-561; advance online publication, September 7, 2017; doi:10.1038/gt.2017.77

The importance of international collaboration for rare diseases research: a European perspective Open  

D Julkowska, C P Austin, C M Cutillo, D Gancberg, C Hager, J Halftermeyer, A H Jonker, L P L Lau, I Norstedt, A Rath, R Schuster, E Simelyte and S van Weely

Gene Ther 24: 562-571; advance online publication, July 27, 2017; doi:10.1038/gt.2017.29

Gene therapy research in Asia  

H-X Deng, Y Wang, Q-r Ding, D-l Li and Yu-quan Wei

Gene Ther 24: 572-577; advance online publication, September 7, 2017; doi:10.1038/gt.2017.62

Management of neuromuscular diseases and spinal muscular atrophy in Latin America  

S Monges and A L Rosa

Gene Ther 24: 578-580; doi:10.1038/gt.2017.68

The state of gene therapy research in Africa, its significance and implications for the future  

P Arbuthnot, M B Maepa, A Ely and M S Pepper

Gene Ther 24: 581-589; advance online publication, July 27, 2017; doi:10.1038/gt.2017.57

Developing gene and cell therapies for rare diseases: an opportunity for synergy between academia and industry  

F Mavilio

Gene Ther 24: 590-592; advance online publication, May 25, 2017; doi:10.1038/gt.2017.36

Delivering advanced therapies: the big pharma approach  

J Tarnowski, D Krishna, L Jespers, A Ketkar, R Haddock, J Imrie and S Kili

Gene Ther 24: 593-598; advance online publication, September 7, 2017; doi:10.1038/gt.2017.65

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Compilation of online resources of relevance to ‘Spinraza and advanced therapies: a stakeholder special’ issue of Gene Therapy  

V Prakash and R J Yáñez-Muñoz

Gene Ther 24: 599; doi:10.1038/gt.2017.61