Parent Project Muscular Dystrophy (PPMD) announced today plans to award Dr. Denis Guttridge of The Ohio State University with a $48,000 grant for his work in…
Heart issues don't just affect some people with Duchenne; they affect all people with Duchenne. What Dr. Guttridge and his team at Ohio State are doing is anticipating the challenges future therapies may face in treating these issues of the heart. To end Duchenne we will have to outsmart the disease progression. Dr. Guttridge is doing just that, by understanding the role NF-kB plays in heart failure. PPMD is so grateful to the work being done at institutes like The Ohio State University and we are proud to support Dr. Guttridge.
BioMarin is providing a further update to the Duchenne Community in light of recent announcements of withdrawal of the EMA marketing application and discontin…
BioMarin has provided a further update to the Duchenne Community in light of recent announcements of withdrawal of the EMA marketing application and discontinuation of clinical and regulatory development of drisapersen (BMN 051) and follow-on products BMN 044, BMN 045 and BMN 053.
Drug Has Fast Track Status, Orphan Drug Designation and Rare Pediatric Disease Designation for Duchenne Muscular Dystrophy Northbrook, Ill. – June 14, 2016 – Marathon Pharmaceuticals, LLC, a biopharmaceutical company developing treatments for rare diseases, today announced it has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for the …
This New Drug Application (NDA) submission starts a process that we hope will result in broad access to deflazacort for all of those living with Duchenne who need it.
The FDA has requested that Sarepta provide dystrophin data from biopsies already obtained from the ongoing confirmatory study of eteplirsen (PROMOVI), as part of its ongoing evaluation of the eteplirsen New Drug Application (NDA). We are hopeful this will be the final request for additional data and result in a decision soon.
PPMD will be live streaming sessions from our 2016 Annual Connect Conference, June 26 - 28. Our DuchenneConnect team will also be live tweeting updates #PPMDconnect
Unable to attend this year's Annual Connect Conference in Orlando? Join us online! PPMD will be live streaming most general sessions and our DuchenneConnect team will be live tweeting updates from many of the research presentations using the hashtag #PPMDconnect.
You’ve seen the headlines and heard the name, now find out more about the latest in gene therapy, CRISPR/Cas9, and what these technologies could mean for Duch…
You’ve seen the headlines and heard the name, now find out more about the latest in gene therapy, CRISPR/Cas9, and what these technologies could mean for Duchenne during three different opportunities this summer that promise to provide an in-depth analysis.
Genetic counselors, whether they are part of PPMD's DuchenneConnect team or part of your local care team, are an excellent resource to consider when you have…
Genetic Testing for Duchenne & Becker: When is re-testing needed? Is there a certain year cut-off to consider re-testing? What resources are available? Read this informative blog from one of PPMD's DuchenneConnect genetic counselors to learn more about the situations that might warrant re-testing.
Summit Therapeutics PLC today announced enrollment of the first patient in their PhaseOut DMD clinical trial in the UK. They continue to expect the US sites to begin enrolling in the third quarter of this year. PhaseOut DMD is a small, open-label trial in which hopes to see that ezutromid (SMT C1100) is able to modify utrophin in the muscles to substitute for dystrophin. We look forward to hearing more from Summit at PPMD's 2016 Connect Conference in Orlando, June 26-29!
The promise of gene therapy continues to build momentum, in Duchenne and also in other conditions. Much has been learned about the immunological and delivery c…
Our community has been interested in the potential of gene therapy as a treatment for Duchenne for many years now. Dr. Guy Odom from the University of Washington, in anticipating potential barriers in the functionality of gene therapy, is addressing a possible immune response head on. PPMD is proud to support Dr. Odom and we are hopeful that his work will help make gene therapy in Duchenne more effective and long lasting.
Santhera Pharmaceuticals announced today that additional data from the pivotal phase III trial (DELOS) were published online as an article in press in Neuromus…
Pulmonary issues, and optimizing pulmonary care for people with Duchenne, has been a longtime priority for PPMD and is a significant part of our extensive care initiatives. We are encouraged by the reported data and we are hopeful that Raxone will become one of the tools in the arsenal that will end Duchenne.
mt is a cross-disciplinary biomedical journal devoted to publishing the most exciting advances in pharmacology and therapeutics, as they pertain to advances in translational and clinical medicine. It is recognized as one of the most prestigious journals in the field. With an impact factor of 6.825*, mt ranks in the top 4.2% of scientific journals in the latest Science Citation Index. Published monthly online and in print.
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