Gene-correction therapy suffers setback but is still seen as promising

09/25/2013 | New York Times (tiered subscription model), The

Anecdotal reports from patients and data from small clinical trials have raised hopes that experimental drugs for Duchenne muscular dystrophy based on a new genetic technique called exon skipping could ultimately lead to a treatment. Drisapersen, an exon-skipping drug being developed by GlaxoSmithKline and Prosensa, failed to beat a placebo in preserving muscle function in boys with the disease in a large trial, but the idea of correcting faulty genes remains promising. "We are fully confident in the exon-skipping technology as a viable platform to develop a treatment for Duchenne," said Debra Miller, president of the advocacy group CureDuchenne.

View Full Article in:

New York Times (tiered subscription model), The

Published in Briefs:

SmartBrief Job Listings for Health Care

Job Title Company Location
Chief Financial Officer
B. E. Smith
Miramar, FL
Claims Director
PacificSource
Springfield, OR
Consultant
Attest Health Care Advisors
Nationwide, SL_Nationwide
Biotechnology/Pharmaceutical Patent Attorney
Coats and Bennett PLLC
Cary, NC
Sr. Regulatory Specialist, Biotech Center of Expertise
BASF, The Chemical Co.
San Diego, CA