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Download: PPMD on benefit-risk assessments in rare disorders

Download: PPMD on benefit-risk assessments in rare disorders | Duchenne 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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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
ParentProjectMD's insight:

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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Prosensa Announces 1st Quarter 2014 Financial Results and Recent Corporate Developments (NASDAQ:RNA)

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Prosensa Holding N.V. to Webcast Conference Call Discussing 1st Quarter 2014 Financial Results and Corporate Update on May 20, 2014

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Prosensa Provides Update on Drisapersen

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Re-dosing plans are underway and 96-week data from an open-label extension study of drisapersen for the treatment of DMD presented at the American Academy of Neurology (AAN)

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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 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…
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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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NCATS Announces Funding Opportunities to Repurpose Drug Candidates from Industry

NCATS Announces Funding Opportunities to Repurpose Drug Candidates from Industry | Duchenne Research | Scoop.it
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PTC Therapeutics Reports First Quarter Financial Results and Provides Corporate Update

PTC Therapeutics Reports First Quarter Financial Results and Provides Corporate Update | Duchenne Research | Scoop.it
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Prosensa Patient Group Communication April 2014

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The latest update from Prosensa regarding re-dosing drisapersen and the regulatory path forward.

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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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