Duchenne Action Month Needs YOU!
This September, we are asking you, our community, to participate in Duchenne Action Month in order to create awareness, raise money, and make an impact in the fight to #endDuchenne. Check out 10 simple and powerful actions you can take to make an impact this September: www.parentprojectmd.org/actionmonth (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - August 22, 2017 Category: Neurology Source Type: news
[Webinar] Gene Therapy for Duchenne - August 2017
Last week, PPMD hosted a webinar focused on understanding the different therapies that are being developed that are commonly referred to as gene therapy, including micro-dystrophin and CRISPR/Cas9, how are they similar and different, and what are the challenges and limitations for each of them. If you missed it, the recording of the webinar is now available!
COMING SOON: This fall PPMD will be hosting additional webinars with companies and institutions who are developing gene therapies and/or CRISPR/Cas9 for Duchenne. Stay tuned for more details! (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - August 22, 2017 Category: Neurology Source Type: news
How You Can Participate in the Ataluren Ad Comm
The FDA has announced the scheduling of a September 28th Advisory Committee Meeting for PTC ’s therapy ataluren. Whether submitting written testimony and/or attending the Ad Comm in person, there are multiple ways for Duchenne community members in the U.S. and abroad to have an impact and share your personal experience with the Advisory Committee reviewing this product. Visit our blog to learn how you can participate. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - August 22, 2017 Category: Neurology Source Type: news
PPMD's Bone Health Workshop Published
Bone health has long been a confusing and often neglected aspect of Duchenne care. There is very little knowledge of underlying bone health in Duchenne, which is further complicated by the possible effects of glucocorticoids on both bone development and bone density/strength. We applaud the incredible work of this group of experts who participated in PPMD's Bone Health Workshop, and hope that, with increased awareness, further work to prevent osteoporosis and enhance bone health in people living with Duchenne will continue. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - August 18, 2017 Category: Neurology Source Type: news
PPMD's Every Single [One] Tour: Charleston, WV
Registration for the next stop on PPMD's Every Single [One] Tour is now open! Combining each of the pillars that make up PPMD's mission -- To #EndDuchenne -- the Every Single [One] Tour brings free day-long meetings featuring updates on research, advocacy, and care to the Duchenne community across the country. We hope to see you in Charleston, WV on September 9! (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - August 17, 2017 Category: Neurology Source Type: news
Understanding Clinical Trials in Duchenne
Did you know that there are over 50 clinical trials going on in the US focused on Duchenne& Becker? While having so many clinical trials is exciting, it can also be overwhelming when you are trying to make a decision about if you want to be in a study and if so, which study is the best fit for you and your family. Learn more about the current clinical trial landscape& the PPMD resources that can help you explore clinical trial options. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - August 7, 2017 Category: Neurology Source Type: news
Webinar Recording: Nationwide Children ’s Hospital Carrier Study
PPMD recently hosted a webinar for a discussion on the Nationwide Children ’s Hospital Carrier Study. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - May 30, 2017 Category: Neurology Source Type: news
FDA Pediatric Advisory Committee Unanimously Votes YES — Now Moves to Commissioner
Today ’s Pediatric Advisory Committee Meeting resulted in a unanimous decision from committee members to recommend the use of in-dwelling ports in Sarepta's ESSENCE clinical trial. A final decision will be announced once the Commissioner has reviewed the summary report. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - May 18, 2017 Category: Neurology Source Type: news
Deciphering What We Know About EMFLAZA
(Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - May 15, 2017 Category: Neurology Source Type: news
ACTION ALERT: Urge Your Senators to Sign the FY18 Duchenne Funding Letter
(Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - May 15, 2017 Category: Neurology Source Type: news
In Honor of Moms
This Mother ’s Day, thank someone for their role in the fight to end Duchenne. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - May 10, 2017 Category: Neurology Source Type: news
Webinar Recording: Results from Phase I/II HOPE Clinical Trial of CAP-1002
PPMD, Coalition Duchenne, and Capricor recently hosted a webinar to hear a community update on the six-month results from Capricor's randomized Phase I/II HOPE Clinical Trial of CAP-1002. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - May 9, 2017 Category: Neurology Source Type: news
PPMD's Response to PTC Announcement of EMFLAZA (deflazacort) Pricing & Access
Today, PTC Therapeutics provided a community update on EMFLAZA (deflazacort) which they acquired earlier in the year from Marathon Pharmaceuticals. PPMD will continue to monitor and remain engaged in the access environment in order to understand the impact of every new approval, including EMFLAZA, and will update our online Access Resource as information becomes available. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - May 8, 2017 Category: Neurology Source Type: news
Meet the Race to End Duchenne .1K!
September is Duchenne Action Month, and now is the time for you to start planning an event than can raise awareness and money, while providing people with an amazing experience that is fun and rewarding …the Race to End Duchenne .1K! (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - May 5, 2017 Category: Neurology Source Type: news
Webinar Recording: Givinostat in Duchenne
PPMD and Italfarmaco recently hosted a webinar to discuss Italfarmaco's investigational drug Givinostat and how it works in Duchenne. (Source: Parent Project Muscular Dystrophy)
Source: Parent Project Muscular Dystrophy - May 1, 2017 Category: Neurology Source Type: news
