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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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Sarepta Enters into Partnership with Flagship Biosciences to Digitally Automate the Measurement of Dystrophin, a Key Therapeutic Efficacy Marker for Muscular Dystrophy

Sarepta Enters into Partnership with Flagship Biosciences to Digitally Automate the Measurement of Dystrophin, a Key Therapeutic Efficacy Marker for Muscular Dystrophy | Duchenne Research | Scoop.it
New technology designed to speed dystrophin measurement while ensuring consistencyAutomation of dystrophin measurement will aid development of Sarepta’s Duchenne muscular dystrophy candidates
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PTC Therapeutics Reports Second Quarter Financial Results and Provides Corporate Update (NASDAQ:PTCT)

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PTC Therapeutics Receives Conditional Approval in the European Union for Translarna™ For the Treatment of Nonsense Mutation Duchenne Muscular Dystrophy (NASDAQ:PTCT)

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The European Commission has granted accelerated approval for Translarna within the European Union, as predicted based on an earlier recommendation by the CHMP.

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Sarepta Therapeutics to Announce Second Quarter 2014 Financial Results and Corporate Update on August 7, 2014

Sarepta Therapeutics to Announce Second Quarter 2014 Financial Results and Corporate Update on August 7, 2014 | Duchenne Research | Scoop.it
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PTC Therapeutics to Host Conference Call to Discuss Second Quarter 2014 Financial Results (NASDAQ:PTCT)

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PTC Therapeutics announces expanded access program for Translarna™ (ataluren)

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International Study on the Burden of Duchenne Published in Neurology

International Study on the Burden of Duchenne Published in Neurology | Duchenne Research | Scoop.it
In the first international study of its kind, researchers have found that there are many different costs accompanying a rare condition such as Duchenne muscula…
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Akashi Therapeutics Receives Fast Track Designation for HT-100 from FDA for the Treatment of Duchenne Muscular Dystrophy

Akashi Therapeutics Receives Fast Track Designation for HT-100 from FDA for the Treatment of Duchenne Muscular Dystrophy | Duchenne Research | Scoop.it
From BioPortfolio: Akashi Therapeutics, Inc., announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to the company�s most adv...
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Prosensa gets an FDA green light for its once-failed DMD drug

Prosensa gets an FDA green light for its once-failed DMD drug | Duchenne Research | Scoop.it
After a late-stage failure and a kiss-off from partner GlaxoSmithKline, Prosensa and its innovative treatment for Duchenne muscular dystrophy seemed to be on a downward slope.
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Funding Opportunities-FY14 Duchenne Muscular Dystrophy Research Program, Congressionally Directed Medical Research Programs (CDMRP), US DoD

Funding Opportunities-FY14 Duchenne Muscular Dystrophy Research Program, Congressionally Directed Medical Research Programs (CDMRP), US DoD | Duchenne Research | Scoop.it
ParentProjectMD's insight:

Two DMDRP PAs have been posted on the CDMRP website.

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PTC Therapeutics Receives Positive Opinion from CHMP for Translarna (Ataluren)

PTC Therapeutics Receives Positive Opinion from CHMP for Translarna (Ataluren) | Duchenne Research | Scoop.it
Read the press release from PTC Therapeutics:
The first treatment for the underlying cause of Duchenne muscular dystrophy

SOUTH PLAINFIELD, NJ – May 23, 2014…
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Webinar: The Patient’s Understanding of Benefit Risk

This webinar will serve to answer several key questions: 

- How do we balance the need for timely access new medicines and benefit risk? 
- Who ultimately should chose what level of uncertainty is acceptable? 
- What are good examples of tools and methods for making informed benefit risk decisions with increased uncertainty? 

Featuring: 
- Andrea Beyer, National Expert Benefit Risk Assessment, European Medicines Agency 
- Yann Le Cam, Chief Executive Officer at EURORDIS 
- Alastair Kent, Director of Genetic Alliance UK 
- Donald Singer, Secretary, European Association of Clinical Pharmacology and Therapeutics 

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SMT C1100 Preliminary Results from Phase 1B Clinical Trial for Treatment of Duchenne

SMT C1100 Preliminary Results from Phase 1B Clinical Trial for Treatment of Duchenne | Duchenne Research | Scoop.it
In the first study of a utrophin up-regulating drug in Duchenne, British biotech company Summit PLC reports that its drug SMT C1100 showed a good safety profil…
ParentProjectMD's insight:

In the first study of a utrophin up-regulating drug in Duchenne, British biotech company Summit PLC reports that its drug SMT C1100 showed a good safety profile in twelve boys with Duchenne. Plasma levels of the drug were variable and the company speculates that diet and the course of the disease may be the cause.

The preliminary trial data will be reviewed further by Summit and is expected to lead to a revision of future clinical trial plans in order to determine the optimal way, either through dietary means or drug formulation changes, to address the drug uptake differences between Duchenne patients and healthy volunteers. The next patient study is now expected to start in Q4 2014.

 

Summit will host a webinar next week to discuss this news and answer your questions - more details to come. For now though, you can submit your questions to info@parentprojectmd.org, RE: SUMMIT.

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Prosensa Announces 2nd Quarter 2014 Financial Results and Recent Corporate Developments (NASDAQ:RNA)

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Sarepta Therapeutics Announces Second Quarter 2014 Financial Results and Recent Corporate Developments

Sarepta Therapeutics Announces Second Quarter 2014 Financial Results and Recent Corporate Developments | Duchenne Research | Scoop.it
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Prosensa Holding N.V. to Webcast Conference Call Discussing 2nd Quarter 2014 Financial Results and Corporate Update on August 12, 2014 (NASDAQ:RNA)

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Exon-Skipping Trial Updates from Sarepta & Prosensa

Exon-Skipping Trial Updates from Sarepta & Prosensa | Duchenne Research | Scoop.it
In the wake of promising exon-skipping trial announcements earlier this summer, PPMD has been fielding many questions from the community about upcoming trial…
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Sarepta Therapeutics Inc - Investors - News Release

Sarepta Therapeutics Inc - Investors - News Release | Duchenne Research | Scoop.it
Sarepta Therapeutics Reports Long-Term Outcomes Through 144 Weeks from Phase IIb Study of Eteplirsen in Duchenne Muscular Dystrophy
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Summit presents new data from Phase 1b clinical trial of SMT C1100 for treatment of Duchenne

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Akashi Therapeutics Presents Positive Clinical Data on HT-100 in Patients with Duchenne

Akashi Therapeutics Presents Positive Clinical Data on HT-100 in Patients with Duchenne | Duchenne Research | Scoop.it
Preliminary Clinical Data from Ongoing Phase 1b/2a Clinical Program Presented at New Directions in Biology and Disease of Skeletal Muscle 6th Biennial Conference Cambridge, Mass.—July 3, 2014—Akashi Therapeutics, Inc., a...
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PTC THERAPEUTICS APPOINTS RONALD C. RENAUD, JR. TO BOARD OF DIRECTORS

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Prosensa Announces A Regulatory Path Forward for Drisapersen as a Potential Treatment for Duchenne

Prosensa Announces A Regulatory Path Forward for Drisapersen as a Potential Treatment for Duchenne | Duchenne Research | Scoop.it
Prosensa Plans to Submit a New Drug Application to the FDA this Year; Dialogue with EMA continues with intent to seek approval

More good news from the FDA! Th…
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A Good Day for Duchenne

A Good Day for Duchenne | Duchenne 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.     

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Filippo Buccella's curator insight, May 23, 7:28 AM

Great Great day! Agree!

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Drisapersen Regulatory Discussions Ongoing & Re-Dosing on Track

Drisapersen Regulatory Discussions Ongoing & Re-Dosing on Track | Duchenne Research | Scoop.it
Below you can find the latest update from Prosensa regarding a potential regulatory path forward for drisapersen.Regulatory discussions ongoing & re-do…
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Sarepta Therapeutics Inc - Investors - News Release

Sarepta Therapeutics Inc - Investors - News Release | Duchenne Research | Scoop.it
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Sarepta Therapeutics Announces Agreement for Acquisition of Manufacturing Facility in Massachusetts


State-of-the-art 60,000 square foot facility enhances internal manufacturing capability

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