Duchenne Muscular Dystrophy Research
84.6K views | +31 today
Follow
 
Scooped by ParentProjectMD
onto Duchenne Muscular Dystrophy Research
Scoop.it!

A Good Day for Duchenne

A Good Day for Duchenne | Duchenne Muscular Dystrophy Research | Scoop.it
EMA grants Conditional Approval for Ataluren
 
Ataluren, PTC Therapeutics’ nonsense suppression drug received Conditional Approval. This is a big moment for Du…
ParentProjectMD's insight:

Ataluren, PTC Therapeutics’ nonsense suppression drug received Conditional Approval. This is a big moment for Duchenne, a landmark decision and a sign of hope for all. While Ataluren is targeted at a small subset of patients (13% +/-), the conditional approval is a sign that the EMA worked very hard to understand Duchenne, that they recognize this unmet need, the progressive and debilitating process of Duchenne and the urgent need to treat.     

more...
Filippo Buccella's curator insight, May 23, 2014 10:28 AM

Great Great day! Agree!

Duchenne Muscular Dystrophy Research
Your new post is loading...
Your new post is loading...
Scooped by ParentProjectMD
Scoop.it!

Sarepta Update for the Duchenne Community Regarding ESSENCE Study

Sarepta Update for the Duchenne Community Regarding ESSENCE Study | Duchenne Muscular Dystrophy Research | Scoop.it
Sarepta has shared an update for the Duchenne community regarding recent changes to the Phase III confirmatory clinical study, ESSENCE (Study 4045-301), as p…
ParentProjectMD's insight:
Sarepta has shared an update for the Duchenne community regarding recent changes to the Phase III confirmatory clinical study, ESSENCE (Study 4045-301).
more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

PPMD Awards Dr. Denis Guttridge, The Ohio State University Grant to Support Cardiomyopathy Therapy for Duchenne

PPMD Awards Dr. Denis Guttridge, The Ohio State University Grant to Support Cardiomyopathy Therapy for Duchenne | Duchenne Muscular Dystrophy Research | Scoop.it
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…
ParentProjectMD's insight:
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.
more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

Santhera's Marketing Authorization Application for Raxone® in Duchenne Muscular Dystrophy (DMD) Validated by the European Medicines Agency

more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

BioMarin Update for the Duchenne Community

BioMarin Update for the Duchenne Community | Duchenne Muscular Dystrophy Research | Scoop.it
BioMarin is providing a further update to the Duchenne Community in light of recent announcements of withdrawal of the EMA marketing application and discontin…
ParentProjectMD's insight:
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.
more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

Marathon Pharmaceuticals Announces Submission of Deflazacort New Drug Application to the FDA - Marathon Pharmaceuticals, LLC

Marathon Pharmaceuticals Announces Submission of Deflazacort New Drug Application to the FDA - Marathon Pharmaceuticals, LLC | Duchenne Muscular Dystrophy Research | Scoop.it
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 …
ParentProjectMD's insight:
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.
more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

PPMD & BIO Release New Report on the Development of Disease-Specific Patient Preference Studies

PPMD & BIO Release New Report on the Development of Disease-Specific Patient Preference Studies | Duchenne Muscular Dystrophy Research | Scoop.it
Today, the Biotechnology Innovation Organization (BIO) and Parent Project Muscular Dystrophy (PPMD) released a new report, “Key Considerations for Developing…
more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

Sarepta Therapeutics Announces FDA Request For Dystrophin Data Prior To Making A Decision on Eteplirsen NDA

Sarepta Therapeutics Announces FDA Request For Dystrophin Data Prior To Making A Decision on Eteplirsen NDA | Duchenne Muscular Dystrophy Research | Scoop.it
ParentProjectMD's insight:
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.
more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

BioMarin Discontinues Clinical Development of Drisapersen

BioMarin Discontinues Clinical Development of Drisapersen | Duchenne Muscular Dystrophy Research | Scoop.it
Today is June 1 and I know many of us feel like 2016 has beat up this community already. With every small victory, we face another major setback.
 
As you ha…
more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

Sarepta Announces FDA Will Not Complete the Review of the Eteplirsen NDA By The PDUFA Date

Sarepta Announces FDA Will Not Complete the Review of the Eteplirsen NDA By The PDUFA Date | Duchenne Muscular Dystrophy Research | Scoop.it
The FDA has notified Sarepta that they are continuing their review of eteplirsen and will not be able to complete their work by the Prescription Drug User Fee…
more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

[Webinar] MissionDMD: FibroGen’s Anti-Fibrosis Program - May 2016

On May 18, 2016, PPMD and FibroGen hosted a webinar to discuss FibroGen’s investigational drug FG-3019 (which is an anti-CTGF monoclonal antibody) and th
more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

WAVE Life Sciences to Advance Next-Generation Nucleic Acid Therapies to Address Unmet Need in Duchenne Muscular Dystrophy

WAVE Life Sciences to Advance Next-Generation Nucleic Acid Therapies to Address Unmet Need in Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy Research | Scoop.it
WAVE Life Sciences Ltd. (NASDAQ: WVE) today reaffirmed its commitment to advance next-generation nucleic acid therapies to address the significant unm
ParentProjectMD's insight:
Share your insight
more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

Join the 2016 #PPMDConnect Conference Online!

Join the 2016 #PPMDConnect Conference Online! | Duchenne Muscular Dystrophy Research | Scoop.it
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
ParentProjectMD's insight:
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.
more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

Pluristem Reports Data Showing PLX-PAD Cells Effective in Treating Duchenne Muscular Dystrophy

more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

Understanding Gene Therapy & CRISPR/Cas9

Understanding Gene Therapy & CRISPR/Cas9 | Duchenne Muscular Dystrophy Research | Scoop.it
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…
ParentProjectMD's insight:
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.
more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

Genetic Testing for Duchenne & Becker: When Is Re-Testing Needed?

Genetic Testing for Duchenne & Becker: When Is Re-Testing Needed? | Duchenne Muscular Dystrophy Research | Scoop.it
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…
ParentProjectMD's insight:
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.
more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

First Patient Enrolled in Summit’s PhaseOut DMD, a Phase 2 Clinical Trial of Ezutromid in Boys With DMD

ParentProjectMD's insight:
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!
more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

PPMD Awards University of Washington Grant to Support Technology to Help Optimize Gene Therapy

PPMD Awards University of Washington Grant to Support Technology to Help Optimize Gene Therapy | Duchenne Muscular Dystrophy Research | Scoop.it
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…
ParentProjectMD's insight:
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.
more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

Catabasis Pharmaceuticals and Parent Project Muscular Dystrophy to Host a Webinar on MoveDMDSM: A Clinical Trial of Edasalonexent (CAT-1004) in Boys with Duchenne Muscular Dystrophy

Catabasis Pharmaceuticals and Parent Project Muscular Dystrophy to Host a Webinar on MoveDMDSM: A Clinical Trial of Edasalonexent (CAT-1004) in Boys with Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy Research | Scoop.it
Catabasis and Parent Project Muscular Dystrophy (PPMD) will host a webinar “MoveDMD: A Clinical Trial of Edasalonexent (CAT-1004) in Boys with D
ParentProjectMD's insight:
Share your insight
more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

New Data from Santhera’s Phase III Trial (DELOS) in Duchenne Published in Neuromuscular Disorders

New Data from Santhera’s Phase III Trial (DELOS) in Duchenne Published in Neuromuscular Disorders | Duchenne Muscular Dystrophy Research | Scoop.it
Santhera Pharmaceuticals announced today that additional data from the pivotal phase III trial (DELOS) were published online as an article in press in Neuromus…
ParentProjectMD's insight:
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.
more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

Santhera Files Marketing Authorization Application in the European
Union for Raxone®
for the Treatment of Duchenne Muscular Dystrophy

more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

Funding Opportunities-FY15 Duchenne Muscular Dystrophy Research Program, Congressionally Directed Medical Research Programs (CDMRP), US DoD

more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

Molecular Therapy - A Phase 1/2a Follistatin Gene Therapy Trial for Becker Muscular Dystrophy

Molecular Therapy - A Phase 1/2a Follistatin Gene Therapy Trial for Becker Muscular Dystrophy | Duchenne Muscular Dystrophy Research | Scoop.it
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.
more...
No comment yet.