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Halo HT-100 Update

Halo HT-100 Update | Duchenne Research | Scoop.it
We are heartbroken to learn more discouraging news about another Duchenne therapy today, HT-100 from Halo Therapeutics. This community continues to face tremen…
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Scientists embark on unprecedented effort to connect millions of patient medical records

Scientists embark on unprecedented effort to connect millions of patient medical records | Duchenne Research | Scoop.it
As researchers embark on a project to connect 30 million patient records, questions about privacy arise.
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Exon-Skipping Therapy: A Roadblock, Detour, or Bump in the Road?

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Commentary by Eric Hoffman and Beth McNally.

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PTC THERAPEUTICS EXPANDS INTERNATIONAL COMMERCIAL TEAM (NASDAQ:PTCT)

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Prothelia and the University of Nevada, Reno Enter Into Exclusive Strategic Agreements with Alexion for Catastrophic, Ultra-Rare Neuromuscular... | NEWS.GNOM.ES

Prothelia and the University of Nevada, Reno Enter Into Exclusive Strategic Agreements with Alexion for Catastrophic, Ultra-Rare Neuromuscular... | NEWS.GNOM.ES | Duchenne Research | Scoop.it
MILFORD, Mass. and RENO, Nev. , Feb
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Sarepta Therapeutics Announces Eteplirsen Demonstrates Stability on Pulmonary Function Tests through 120 Weeks in Phase IIb Study in Duchenne Muscular Dystrophy

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Prosensa Reports Initial Findings from the Further Clinical Data Analyses of Drisapersen for the Treatment of Duchenne Muscular Dystrophy

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Sarepta Therapeutics Names Art Krieg, M.D., Chief Scientific Office

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Halo HT-100 Update

Halo HT-100 Update | Duchenne Research | Scoop.it
We are heartbroken to learn more discouraging news about another Duchenne therapy today, HT-100 from Halo Therapeutics. This community continues to face tremen…
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Ataluren Clinical Data Demonstrate an Increase in Dystrophin Expression in Duchenne Patients

PTC Therapeutics, Inc., today announced the publication of data in PLOS ONE demonstrating that nonsense mutation Duchenne muscular dystrophy (nmDMD) patients treated with ataluren, an investigational new drug, experienced an increase in dystrophin expression. These data were obtained from PTC's Phase 2a open-label trial of ataluren in which change in full-length dystrophin expression, as assessed by immunofluorescent staining, was the primary endpoint.

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Summit Corp, Oxford University in Duchenne alliance - Stock Market Wire

Summit Corp, Oxford University in Duchenne alliance - Stock Market Wire | Duchenne Research | Scoop.it
Proactive Investors UK
Summit Corp, Oxford University in Duchenne alliance
Stock Market Wire
StockMarketWire.com - Summit Corp, a drug discovery and development company advancing therapies for Duchenne Muscular Dystrophy (DMD) and C.
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PTC THERAPEUTICS REPORTS THIRD QUARTER 2013 FINANCIAL AND CORPORATE RESULTS

PTC THERAPEUTICS REPORTS THIRD QUARTER 2013 FINANCIAL AND CORPORATE RESULTS | Duchenne Research | Scoop.it

PTC Therapeutics, Inc., a biopharmaceutical company focused on the discovery and development of orally administered, proprietary small molecule drugs that target post-transcriptional control processes, today announced financial and corporate results for the quarter ended September 30, 2013.

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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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GSK Q&A for Patient Advocacy Groups: October 28, 2013

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Prosensa to Appoint Michael Wyzga to Supervisory Board

Prosensa Holding N.V. (NASDAQ: RNA), the Dutch biopharmaceutical company focusing on rare diseases with a high unmet medical need, today announced that Michael S. Wyzga has been nominated for appointment to its Supervisory Board at the next shareholders' meeting, scheduled for June 17, 2014.

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Prosensa Announces Drisapersen 48-Week Data and Recent Corporate Developments

Prosensa Announces Drisapersen 48-Week Data and Recent Corporate Developments | Duchenne Research | Scoop.it
Prosensa just released their 48-week data for drisapersen, as well as an update on recent corporate developments. Both press releases are included below.
PPMD…
ParentProjectMD's insight:

Prosensa just released their 48-week data for drisapersen, as well as an update on recent corporate developments. Both press releases are included.

PPMD has been talking with Prosensa and will be hosting a webinar with UPPMD later this month. We will be asking you for questions to pass along to Prosensa prior to the webinar. More to come soon.

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Prosensa Outlines Next Steps for Drisapersen

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Nanoparticles deliver drug to mice with muscular dystrophy - Futurity

Nanoparticles deliver drug to mice with muscular dystrophy - Futurity | Duchenne Research | Scoop.it
When treated with nanoparticles loaded with an immunosuppressive drug, mice with muscular dystrophy showed improved strength and heart function.
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PTC THERAPEUTICS PROVIDES UPDATE ON CHMP OPINION FOR CONDITIONAL APPROVAL OF ATALUREN FOR NONSENSE MUTATION DUCHENNE

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Sarepta Therapeutics Announces Eteplirsen Demonstrates Continued Stability on Walking Test through 120 Weeks in Phase IIb Study in Duchenne Muscular Dystrophy

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Prosensa Regains Rights to Drisapersen From GSK and Retains Rights to All Other Programs for the Treatment of Duchenne Muscular Dystrophy (DMD) (NASDAQ:RNA)

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Current Status of Drisapersen Studies

Current Status of Drisapersen Studies | Duchenne Research | Scoop.it
Analyses and assessment of next steps are ongoing
 
PPMD just received the following update from GSK regarding the current status of their drisapersen study:
D…
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Prosensa Announces Extraordinary Shareholders Meeting to Appoint Dr. Georges Gemayel

Prosensa Holding N.V. (NASDAQ: RNA), the Dutch biopharmaceutical company focusing on rare diseases with a high unmet medical need, today announced that an Extraordinary General Meeting of Shareholders will be held on January 23rd, 2014 at 14:00 hrs. CET at the offices of Prosensa Holding N.V., J.H. Oortweg 21, 2333 CH Leiden, The Netherlands. The purpose of the meeting is to appoint Dr. Georges Gemayel, PhD as a new Supervisory Board member. Dr. Georges Gemayel currently serves on several boards, including those of NPS Pharmaceuticals, Orphazyme, Vascular Magnetics and EpiTherapeutics. Previous board memberships include Executive Chairman of the Board at FoldRx, a neurodegenerative disease company that is now a wholly owned subsidiary of Pfizer and a board director at Adolor Corporation, which has since been acquired by Cubist Pharmaceuticals. Dr. Georges Gemayel's operational experience is extensive, having served as President & CEO of Waltham based Altus Pharmaceuticals and most notably as Executive Vice President of Genzyme Corporation from 2003 to 2008, among others.
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New muscular dystrophy treatment shows promise in early study led by Children's National - The Almagest

New muscular dystrophy treatment shows promise in early study led by Children's National - The Almagest | Duchenne Research | Scoop.it
A preclinical study led by researchers at Children’s National Medical Center has found that a new oral drug shows early promise for the treatment of Duchenne muscular dystrophy (DMD).
ParentProjectMD's insight:

This is progress on the ReveraGen drug, a new chemical entity that has been developed as a potential steroid replacement, without the significant side effects of steroids. This is amazing progress and we are hopeful/anxious to see this move into clinical studies.

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Sarepta Therapeutics Announces FDA Considers NDA Filing for Eteplirsen Premature in Light of Recent Competitive Drug Failure and Recent DMD Natural History Data

FDA Questions Dystrophin as a Biomarker Due to Failed Studies of Other Investigational Drugs for DMD; FDA Questions 6-Minute Walk Test Results for Eteplirsen, Suggesting Study Population Should Be Stable Over Two-Year Timeframe Due to Recent Natural History Data; FDA Requests Further Discussion on Endpoints, Design of Confirmatory Clinical Study
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GSK Q&A For Patient Advocacy Groups: October 4, 2013

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