WMS 2013 was full of excitement and interest as results of current clinical trials and early stage research were discussed. Here are some highlights, thank you Abby Bronson for summarizing […]
Dr. Adeline Vulin from Nationwide Children’s Hospital presented the latest data from her work in Dr. Kevin Flanigan’s lab on the new duplication mouse model and the results of exon […]
Here are top line notes from the PRO044 presentation at World Muscle Society, Oct 4. Age of inclusion: 5-16 Mean age: 9 The drug was well tolerated. They saw trace […]
Conference call and live webcast scheduled for 11:00 a.m. ET (5:00 p.m. CET) Tuesday, October 8, 2013 LEIDEN, The Netherlands – October 3, 2013 – Prosensa Holding N.V. (NASDAQ: RNA), […]
We wanted to let Duchenne families know about a new online resource on exon skipping from Sarepta Therapeutics. Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a developer of innovative RNA-based therapeutics, today […]
We are encouraged by the promising Phase IIb data for eteplirsen, an exon-skipping compound, which we hope could be a breakthrough treatment for Duchenne muscular dystrophy. Currently, there is no […]
GlaxoSmithKline (GSK) and Prosensa today announced that GSK’s Phase III clinical study of drisapersen, an investigational antisense oligonucleotide, for the treatment of Duchenne Muscular Dystrophy (DMD) patients with an amenable […]
CureDuchenne has worked closely with biotech and pharmaceutical companies for the past decade with a goal of accelerating treatments for Duchenne muscular dystrophy. CureDuchenne was the first organization to partner […]
Aug 30, 2013 Skipping multiple exons at the same time, by using a combination of antisense oligonucleotides, offers the potential to treat a significant number of Duchenne patients. This […]
