Duchenne Muscular Dystrophy Research
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Prosensa Enrolls 100th Patient to its Natural History Study of Duchenne Muscular Dystrophy (NASDAQ:RNA)

Prosensa Holding N.V. (NASDAQ: RNA), the Dutch biopharmaceutical company focusing on RNA-modulating therapeutics for rare diseases with high unmet need, today announced the successful enrollment of the 100th  patient into the Natural History Study of Duchenne muscular dystrophy (DMD).

The goal of this observational study is to characterize DMD at various stages of progression using the same measures used in ongoing clinical studies, such as the "six minute walk test." No medication is being tested in this study.

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Mallinckrodt Receives FDA Fast-Track Designation for Synacthen® Depot IND Application

Mallinckrodt Receives FDA Fast-Track Designation for Synacthen® Depot IND Application | Duchenne Muscular Dystrophy Research | Scoop.it
CHESTERFIELD, United Kingdom, Aug. 25, 2016 /PRNewswire/ -- Mallinckrodt Receives FDA Fast-Track Designation for Synacthen® Depot IND Application
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Summit Reports Positive Phase 1 Data and Outlines Route to Market Strategy for DMD Candidate Ezutromid

Summit Reports Positive Phase 1 Data and Outlines Route to Market Strategy for DMD Candidate Ezutromid | Duchenne Muscular Dystrophy Research | Scoop.it
Today, Summit Therapeutics plc announced positive results from a Phase 1 trial of a new formulation of ezutromid, which they are calling F6. In this trial, S…
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Today, Summit Therapeutics plc announced positive results from a Phase 1 trial of a new formulation of ezutromid, which they are calling F6. In this trial, Summit showed that F6 is able to be better absorbed in patients than the current formulation of ezutromid, which they are calling F3. Because of these positive results, Summit plans to incorporate F6 into its PhaseOut DMD clinical trial alongside F3. This allows Summit to study the effect of higher concentrations of ezutromid with F6 and evaluate how its safety and efficacy compare with the F3 formulation over the 48 weeks of dosing.
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Study of SRP-4045 and SRP-4053 in DMD Patients - Full Text View - ClinicalTrials.gov

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Sarepta Therapeutics is now recruiting for their exon 45 and exon 53 studies. Visit ClinicalTrials.gov for information on trial sites and inclusion criteria.
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2016 Connect Conference Presentations - Parent Project Muscular Dystrophy

2016 Connect Conference Presentations - Parent Project Muscular Dystrophy | Duchenne Muscular Dystrophy Research | Scoop.it
If you were unable to attend PPMD's 2016 Connect Conference or if there is a session you would like to revisit, most presentations are now available on our website. Thank you to all of our incredible speakers!
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We are excited to share with you presentations from PPMD’s 22nd Annual Connect Conference! If you were unable to attend this year’s Connect Conference, this is your opportunity to review some of the incredibly informative panels and discussions from this year’s meeting. We will continue to update this page over the coming weeks with slides and video content.
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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…
ParentProjectMD's insight:
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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FDA Accepts Marathon Pharmaceuticals’ New Drug Applications for Deflazacort for the Treatment of Duchenne and Grants Priority Review

FDA Accepts Marathon Pharmaceuticals’ New Drug Applications for Deflazacort for the Treatment of Duchenne and Grants Priority Review | Duchenne Muscular Dystrophy Research | Scoop.it
Marathon Pharmaceuticals announced this morning that the U.S. Food and Drug Administration (FDA) has accepted Marathon’s New Drug Applications (NDAs) for the…
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PhaseBio Announces Dosing of First Patients in Two-Part Phase 2a Study of PB1046 in Cardiopulmonary Disorders

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PFIZER AIMS TO BECOME INDUSTRY LEADER IN GENE THERAPY WITH AQUISITION OF BAMBOO THERAPEUTICS, INC. | Pfizer: One of the world's premier biopharmaceutical companies

PFIZER AIMS TO BECOME INDUSTRY LEADER IN GENE THERAPY WITH AQUISITION OF BAMBOO THERAPEUTICS, INC. | Pfizer: One of the world's premier biopharmaceutical companies | Duchenne Muscular Dystrophy Research | Scoop.it
Pfizer Inc. (NYSE:PFE) today announced that it has acquired Bamboo Therapeutics, Inc., a privately held biotechnology company based in Chapel Hill, N.C., focused on developing gene therapies for the potential treatment of patients with certain rare diseases related to neuromuscular conditions and those affecting the central nervous system.
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PTC Therapeutics Provides Regulatory Update on Translarna™

PTC Therapeutics Provides Regulatory Update on Translarna™ | Duchenne Muscular Dystrophy Research | Scoop.it
PTC recently participated in discussions with FDA to discuss the Refuse to File (RTF) letter issued on February 22, 2016 with respect to the company's New Dr…
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Today PTC announced that they have submitted an appeal to escalate continuing discussions about the Refuse to File letter issued earlier this year with respect to the company's New Drug Application (NDA) for Translarna. We strongly believe that PTC and the families in our community that have participated for years in this trial, deserve the opportunity to share the data they have collected and their experiences with this therapy. Join us on August 1 at 11am eastern as we host a webinar with PTC to discuss next steps with the FDA.
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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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