Exon-skipping therapy: A roadblock, detour, or bump in the road?

Eric P. Hoffman; Elizabeth M. McNally

doi:10.1126/scitranslmed.3008873

Exon-skipping therapy: A roadblock, detour, or bump in the road?

Eric P. Hoffman
, Elizabeth M. McNally

Pharmaceutical Sciences

Binghamton University

The University of Chicago

Research output: Contribution to journal › Short survey › peer-review

32 Scopus citations

Abstract

Exon skipping is a promising therapeutic for Duchenne muscular dystrophy patients, but the road to drug approvals is foggy and may require more early-stage derisking and regulatory guidance.

Original language	English
Article number	230fs14
Journal	Science Translational Medicine
Volume	6
Issue number	230
DOIs	https://doi.org/10.1126/scitranslmed.3008873
State	Published - Apr 2 2014

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title = "Exon-skipping therapy: A roadblock, detour, or bump in the road?",

abstract = "Exon skipping is a promising therapeutic for Duchenne muscular dystrophy patients, but the road to drug approvals is foggy and may require more early-stage derisking and regulatory guidance.",

author = "Hoffman, \{Eric P.\} and McNally, \{Elizabeth M.\}",

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T2 - A roadblock, detour, or bump in the road?

AU - Hoffman, Eric P.

AU - McNally, Elizabeth M.

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N2 - Exon skipping is a promising therapeutic for Duchenne muscular dystrophy patients, but the road to drug approvals is foggy and may require more early-stage derisking and regulatory guidance.

AB - Exon skipping is a promising therapeutic for Duchenne muscular dystrophy patients, but the road to drug approvals is foggy and may require more early-stage derisking and regulatory guidance.

UR - https://www.scopus.com/pages/publications/84899120609

U2 - 10.1126/scitranslmed.3008873

DO - 10.1126/scitranslmed.3008873

M3 - Short survey

C2 - 24695683

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VL - 6

JO - Science Translational Medicine

JF - Science Translational Medicine

IS - 230

M1 - 230fs14

ER -

Exon-skipping therapy: A roadblock, detour, or bump in the road?

Abstract

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