CureDuchenne was pleased to host the “Drug Development and Clinical Trial Participation – A Webinar for the Rare Disease Community” on Friday, September 13, 2013. The webinar was an opportunity for parents to learn about the drug development process – from the pre-clinical phase to the clinical phase to the post approval phase and the regulatory process guiding these phases – from industry experts.
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 […]
CureDuchenne, a nonprofit that raises awareness and funds research to find a cure for Duchenne muscular dystrophy, will host the Ì¢âÂÒDrug Development and Clinical Trial Participation Ì¢âÂÛÏ A Webinar for the Rare Disease CommunityÌ¢âÂÌ_ on September 13, 2013 at 2 p.m. EST. The webinar is an opportunity for parents to learn about the drug development process Ì¢âÂÛÏ from the pre-clinical phase to the clinical phase to the post approval phase and the regulatory process guiding these phases Ì¢âÂÛÏ from industry experts.
Yesterday, Fergie – a Grammy award-winning singer/songwriter, and new mom! – joined the ranks of talented and respected celebrities we love. Over the last few years, Clay Matthews, Ryan Getzlaf, […]
Ryan Getzlaf, captain of the Anaheim Ducks, is hosting the third annual Getzlaf Golf Shootout on September 7 and 8 to benefit CureDuchenne, a nonprofit that raises awareness and funds research to find a cure for Duchenne muscular dystrophy. CureDuchenne is pleased to announce that Patriot Environmental Services, the premier provider of comprehensive environmental services in the southwestern United States, is the title sponsor of the Getzlaf Golf Shootout. Patriot is sponsoring the event in memory of Justin Paul Monastesse who lived with Duchenne for more than 25 years and passed on August 1, 2001.
CureDuchenne, a national nonprofit organization that raises awareness and funds research for Duchenne muscular dystrophy, announced today that Fergie, the eight-time Grammy award-winning singer/songwriter, is appearing in a new public service announcement (PSA) campaign on behalf of the organization. The PSA is part of CureDuchenneÌ¢âÂã¢s efforts to raise awareness of Duchenne, the most common and lethal form of muscular dystrophy, and raise funds to develop a robust pipeline of therapies that target the disease from every angle.
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 […]
CureDuchenne’s chief scientific advisor, Dr. Mike Kelly reviewed the recent paper by Annemieke Aartsma-Rus and other researchers. This is complicated but very important science, we attempt here to explain the findings. https://www.fasebj.org/content/early/2013/08/22/fj.13-232025.abstract […]
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 would address one of the major limitations of current antisense therapy, in that the approach is “personalizedâ€ù and designed to skip a single exon.
Specifically, exons 45 to 55 cover the main mutation “hotspotâ€ù of the DMD gene and this area is thought to harbor mutations that are present in more than half of Duchenne patients.
Individuals with this specific 45-55 deletion show almost asymptomatic skeletal muscle involvement or exceptionally mild clinical symptoms and it is this observation that has spurred interest in developing strategies for multi-exon skipping.
This review (https://www.hindawi.com/journals/bmri/2013/402369/), by leading researchers in the field of multiexon skipping, highlights novel findings from a DMD mouse model utilizing systemic multiexon skipping targeting exons 45–55.
The authors highlight the hurdles and limitations impeding the clinical translation of this approach and provide a perspective on the opportunity for exon 45–55 skipping in DMD patients.
Title: Development of Multiexon Skipping Antisense Oligonucleotide Therapy for Duchenne Muscular Dystrophy.
Ref. BioMed Research International, Volume 2013 (2013), https://dx.doi.org/10.1155/2013/402369
Authors: Yoshitsugu Aoki, Toshifumi Yokota, and Matthew J. A. Wood
