To help families and caregivers understand the evolving drug development process, in particular with gene therapies, CureDuchenne, the global leader focused on funding and finding a cure for Duchenne, announced today it will host a free webinar for the Duchenne community.
The Duchenne Clinical Trial and Drug Development: Today and in The Future webinar will take place Wednesday, September 11, 2019 at 2:30 PM (PT) / 5:30 PM (ET). To register, please go to: https://cc.readytalk.com/r/x7znzfd37lft&eom.
“Twenty years ago, there were no clinical trials for Duchenne and parents did not have options. Today, there are many pharmaceutical companies that have joined the race to find viable treatments and cures for Duchenne and there are clinical trials all over the world,” said Debra Miller, founder and CEO of CureDuchenne. “CureDuchenne is dedicated to assisting families navigate the clinical trial options and understand the entire drug development process.”
On the webinar, four leading Duchenne experts will share their inside knowledge about drug development and clinical trials with emphasis on the newest gene therapies. Moderated by Sharon Hesterlee, PhD, executive vice president, portfolio development and head of neuromuscular programs of Asklepios Biopharmaceuticals (AskBio), the webinar will consist of three modules, as follows:
| Module 1: | Drug Development: from Discovery to Approval | |
| Presenter: Peter Marks, MD, PhD, Director, Center for Biologics Research, Food and Drug Administration (FDA) | ||
|
| ||
| Module 2: | Gene Therapy Development Considerations | |
| Presenter: Petra Kaufmann, MD, Vice President, Research and Development, Translational Medicine, AveXis, Inc. | ||
|
| ||
| Module 3: | Maximizing Trial Participation Opportunities | |
| Presenter: Barry Byrne, MD, PhD, Director, UF Powell Gene Therapy Center, and Associate Chair, Pediatrics, University of Florida College of Medicine |
Caregivers can expect to learn about: the drug development pathway, with special emphasis on topics specific to gene therapies; the clinical trial process and recent innovations in trial design; challenges associated with complex manufacturing and the impact they have on availability and price; and how families can best position themselves to participate in trials.
About CureDuchenne
CureDuchenne is the leading nonprofit dedicated to extending and improving the lives of 300,000 boys worldwide who have Duchenne muscular dystrophy, a degenerative disease with no known cure. CureDuchenne uses an entrepreneurial venture philanthropy model to fund impactful research and accelerate human clinical trials. Through its CureDuchenne Cares program, CureDuchenne provides confidence to families seeking valuable resources, trains physical therapists and other health professionals on new standards of care and provides hope, support and empowerment to the Duchenne community. For more information, please visit www.cureduchenne.org.
View source version on businesswire.com: https://www.businesswire.com/news/home/20190828005664/en/
Source: CureDuchenne
View this news release online at:
http://www.businesswire.com/news/home/20190828005664/en
