Duchenne Muscular Dystrophy Research
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Download: PPMD on benefit-risk assessments in rare disorders

Download: PPMD on benefit-risk assessments in rare disorders | Duchenne Muscular Dystrophy Research | Scoop.it
Pat Furlong of Parent Project Muscular Dystrophy kindly shared this whitepaper, ‘Benefit-Risk Assessments in Rare Disorders: The case for therapeutic development
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Total Orphan Drugs covers PPMD's whitepaper. 

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[Webinar] What is CRISPR/Cas9? - July 2016

You’ve seen the headlines and heard the name, now find out more about CRISPR/Cas9 and what this technology could mean for Duchenne. On July 13, 2016 PPM
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Last week PPMD welcomed Dr. Eric Olson from UT Southwestern Medical Center to discuss the basics of gene editing and the potential permanent correction of Duchenne mutations with CRISPR/Cas9. If you missed it live, check out the recording to learn more about this technology and what it could mean for Duchenne!
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Catabasis Quarterly Newsletter: July 2016

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Catabasis provides a quarterly update on their MoveDMD trial (CAT -1004 / edasalonexent), highlighting the open-label extension for the MoveDMD trial and a summary of the findings to date. We appreciate these updates and are proud to have a been an early supporter of this promising therapy!
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BioMarin Update for the Duchenne Community: July 7, 2016

BioMarin Update for the Duchenne Community: July 7, 2016 | Duchenne Muscular Dystrophy Research | Scoop.it
BioMarin has provided a further update to the Duchenne Community following the company's announcement of the withdrawal of the EMA marketing application and di…
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BioMarin has provided a further update to the Duchenne Community following the company's announcement of the withdrawal of the EMA marketing application and discontinuation of clinical and regulatory development of drisapersen (BMN 051) and follow-on compounds BMN 044, BMN 045, and BMN 053.
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NHS England Enables Access to Translarna™ ▼ (ataluren) For Patients with Nonsense Mutation Duchenne Muscular Dystrophy (NASDAQ:PTCT)

NHS England Enables Access to Translarna™ ▼ (ataluren) For Patients with Nonsense Mutation Duchenne Muscular Dystrophy (NASDAQ:PTCT) | Duchenne Muscular Dystrophy Research | Scoop.it

--Important decision allows reimbursed access to Translarna, the first approved therapy to treat the underlying cause of Duchenne muscular dystrophy--

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NHS England enables access to Translarna (ataluren) for people with nonsense mutation Duchenne
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PTC Initiates Phase 2 Clinical Trial of Translarna™ (ataluren) for Treatment of Nonsense Mutation Duchenne Muscular Dystrophy in Pediatric Patients Between the Ages of Two and Five Years

PTC Initiates Phase 2 Clinical Trial of Translarna™ (ataluren) for Treatment of Nonsense Mutation Duchenne Muscular Dystrophy in Pediatric Patients Between the Ages of Two and Five Years | Duchenne Muscular Dystrophy Research | Scoop.it
PTC Therapeutics has announced that they have initiated a Phase 2 clinical trial of Translarna™ (ataluren) for children 2 to 5 years old. Details of site loc…
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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
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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.
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Pluristem Reports Data Showing PLX-PAD Cells Effective in Treating Duchenne Muscular Dystrophy

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

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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!
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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.
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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
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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.
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Pluristem Duchenne Research

Pluristem Reports Data Showing PLX-PAD Cells Effective in Treating Duchenne Muscular Dystrophy
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Pluristem Reports Data Showing PLX-PAD Cells Effective in Treating Duchenne
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Santhera Updates on U.S. Regulatory Filing for Raxone® (idebenone) in Duchenne

Santhera Updates on U.S. Regulatory Filing for Raxone® (idebenone) in Duchenne | Duchenne Muscular Dystrophy Research | Scoop.it
PPMD is deeply disappointed with the FDA’s conclusion regarding Santhera's proposed subpart H (Accelerated Approval) for Raxone (Idebenone). Because individua…
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PPMD is deeply disappointed with the FDA’s conclusion regarding Santhera's proposed subpart H (Accelerated Approval) for Raxone (idebenone). Because individuals in the study were not taking steroids, FDA has recommended Santhera complete the SIDEROS study on individuals on steroids and Raxone (idebenone) prior to submitting an NDA for review. For our community, this means a delay in time that we cannot afford. We are in touch with Santhera about how we can best help moving forward.
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[Upcoming Webinar] What is CRISPR/Cas9?

[Upcoming Webinar] What is CRISPR/Cas9? | Duchenne Muscular Dystrophy Research | Scoop.it
Wednesday, July 13th at 1pm eastern

You’ve seen the headlines and heard the name, now find out more about CRISPR/Cas9 and what this technology could mean for…
ParentProjectMD's insight:
You’ve seen the headlines and heard the name, now find out more about CRISPR/Cas9 and what this technology could mean for Duchenne. PPMD welcomes Dr. Eric Olson from the Department of Molecular Biology at UT Southwestern Medical Center to discuss the basics of gene editing and the potential permanent correction of Duchenne mutations with CRISPR/Cas9.
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Catabasis Pharmaceuticals Announces the Initiation of an Open-Label Extension for the MoveDMD® Trial Studying Edasalonexent (CAT-1004) in Duchenne Muscular Dystrophy

Catabasis Pharmaceuticals Announces the Initiation of an Open-Label Extension for the MoveDMD® Trial Studying Edasalonexent (CAT-1004) in Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy Research | Scoop.it
Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB) today announced the initiation of an open-label extension for the Phase 2 portion (Part B) of the MoveDM
ParentProjectMD's insight:
More progress from Catabasis! Today the company announced the initiation of an open-label extension for the Phase 2 portion (Part B) of the MoveDMD trial studying edasalonexent (CAT-1004) in people with Duchenne.
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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…
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Sarepta has shared an update for the Duchenne community regarding recent changes to the Phase III confirmatory clinical study, ESSENCE (Study 4045-301).
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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.
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Santhera's Marketing Authorization Application for Raxone® in Duchenne Muscular Dystrophy (DMD) Validated by the European Medicines Agency

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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.
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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.
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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…
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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.
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