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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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Sarepta Therapeutics Announces FDA Has Filed Eteplirsen NDA for the Potential Treatment of Duchenne Muscular Dystrophy for Patients Amenable to Exon 51 Skipping (August 25th)

Sarepta Therapeutics Announces FDA Has Filed Eteplirsen NDA for the Potential Treatment of Duchenne Muscular Dystrophy for Patients Amenable to Exon 51 Skipping (August 25th) | Duchenne Research | Scoop.it
ParentProjectMD's insight:

Exciting news from Sarepta Therapeutics! The FDA has completed its filing review and has determined that Sarepta's application is sufficiently complete to permit a substantive review. The Prescription Drug User Fee Act (PDUFA) action date for a decision on the application is February 26, 2016. The FDA has granted eteplirsen Priority Review status, which is designated to drugs that offer benefit over existing therapies, or provide a treatment where no adequate therapy exists.

We anticipate both Sarepta and BioMarin to participate in Advisory Committee (Ad Comm) meetings with FDA in late fall. Don't forget to attend PPMD's Ad Comm Webinar on September 2 so that you and your family can play an active role in helping us get these potential therapies approved:http://on.fb.me/1V8h16Q

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Pfizer Currently Recruiting Participants For Phase 2 Duchenne Muscular Dystrophy Clinical Trial

Pfizer Currently Recruiting Participants For Phase 2 Duchenne Muscular Dystrophy Clinical Trial | Duchenne Research | Scoop.it
Read about Pfizer’s Phase 2 clinical trial on PF-06252616 for Duchenne muscular dystrophy treatment that is currently recruiting participants.
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Sarepta Therapeutics Announces Formation of Strategic & Scientific Advisory Board

Sarepta Therapeutics Announces Formation of Strategic & Scientific Advisory Board | Duchenne Research | Scoop.it

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of innovative RNA-targeted therapeutics, today announced the formation of a Strategic & Scientific Advisory Board (SSAB) with three key appointments:Louis Kunkel, PhD, Beverly Davidson, PhD, and Jeffrey Chamberlain, PhD.

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Different Together: PPMD Announces the Duchenne Regulatory Science Consortium

Different Together: PPMD Announces the Duchenne Regulatory Science Consortium | Duchenne Research | Scoop.it
Signal to Noise
This is a measure used in science and engineering to compare the level of a desired signal (a measured outcome) to the background noise. In a…
ParentProjectMD's insight:

We are thrilled to announce the launch of the Duchenne Regulatory Sciences Consortium (D-RSC) with the Critical Path Institute! Each instance of Duchenne seems to be as unique as the person it affects. The D-RSC will begin by aggregating a wide array of clinical data for analysis by consortium members, with the goal of developing a disease progression model that can be used in the development of new treatments, and reduce the amount of time it takes to get them to people living with Duchenne.
 

Dr. Brenda Wong & Cincinnati Children’s Hospital, PTC Therapeutics, and Santhera Pharmaceuticals are founding members, and we are currently working together to reach a final agreement for Sarepta Therapeutics to join the D-RSC consortium as well. Several additional consortium members will be added soon.

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PPMD's 2015 Connect Conference Resources & Recordings - Part 2 (Friday)

PPMD's 2015 Connect Conference Resources & Recordings - Part 2 (Friday) | Duchenne Research | Scoop.it
Presentations from Day 2 of PPMD’s 21st Annual Connect Conference in Washington, D.C. are now available for download. If you were unable to attend this year’s…
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Presentations from Day 2 of PPMD’s 21st Annual Connect Conference are now available for download! Check out sessions with FDA, representatives from 7 industry partners, and take a few minutes to be inspired when you Meet the PAAC-men.

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Summit Therapeutics Publishes Preclinical Data on Disease Modifying Potential of Utrophin Modulation in DMD

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Key Findings: Neurobehavioral Concerns among Males with Dystrophinopathy Using Population-Based Surveillance Data from the Muscular Dystrophy Surveillance, Tracking, and Research Network

Key Findings: Neurobehavioral Concerns among Males with Dystrophinopathy Using Population-Based Surveillance Data from the Muscular Dystrophy Surveillance, Tracking, and Research Network | Duchenne Research | Scoop.it
Researchers publish new study on mental health among males with Duchenne or Becker muscular dystrophy.
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BioMarin Announces EMA Validates MAA for Drisapersen for Treatment of Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

BioMarin Announces EMA Validates MAA for Drisapersen for Treatment of Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping | Duchenne Research | Scoop.it
SAN RAFAEL, Calif., June 25, 2015 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced the European Medicines Agency (EMA) has validated the Marketing Authorization Application (MAA) for drisapersen for the treatment of Duchenne Muscular Dystrophy amenable to exon 51 skipping. Validation of the MAA confirms that the submission is complete and starts the EMA's standard review process. Day 120 questions will be received on 22 October 2015, leading to a potential CHMP opinion in the first half of 2016 and a European Commission Decision by the third quarter of 2016.
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June 10, 2015: Santhera Update on Pre-NDA Meeting with FDA on Raxone®/Catena® in Duchenne Muscular Dystrophy (DMD)

June 10, 2015: Santhera Update on Pre-NDA Meeting with FDA on Raxone®/Catena® in Duchenne Muscular Dystrophy (DMD) | Duchenne Research | Scoop.it
ParentProjectMD's insight:

Today, Santhera announced plans to meet with FDA officials for a second time to continue discussions around their pre-NDA filing for Raxone®/Catena®. Because of their Fast Track designation, the company is allowed more interaction with the agency. Santhera will be presenting next week at PPMD’s Connect Conference and we look forward to learning more and finding out how PPMD can help.

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BioMarin Submits Drisapersen MAA to EMA for the Treatment of Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

BioMarin Submits Drisapersen MAA to EMA for the Treatment of Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping | Duchenne Research | Scoop.it
SAN RAFAEL, Calif., June 8, 2015 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced today the submission of a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for drisapersen, an investigational antisense oligonucleotide drug candidate for the treatment of the largest subset of Duchenne muscular dystrophy (DMD) amenable to single exon skipping. DMD is the most common fatal genetic disorder diagnosed in childhood, affecting approximately 1 in every 3,500 live male births with about 20,000 new cases diagnosed globally each year. In Europe, it is estimated there are 23,000 boys with Duchenne Muscular Dystrophy, and approximately 3,000 of those would be candidates for drisapersen. In BioMarin's commercial territories, approximately 85 percent of Duchenne patients are located outside of the United States, including Western Europe, Middle East, Eastern Europe, Latin America and Japan.  Western Europe has the largest patient population among those areas, exceeding the United States by around 30 percent.
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SomaLogic Study Published: Another Piece of the Puzzle

SomaLogic Study Published: Another Piece of the Puzzle | Duchenne Research | Scoop.it
Today, SomaLogic and PPMD - together with several other Duchenne research groups - published a significant discovery that explains a technology allowing resea…
ParentProjectMD's insight:

Breakthrough news! Today, SomaLogic and PPMD - together with several other Duchenne research groups - published a significant discovery that explains a technology allowing researchers to accurately measure the individual proteins in very small amounts of blood or other samples. By comparing patient and control samples, identification of critical differences in protein concentrations can be identified rapidly. This means that this data could be utilized as an outcome measure for clinical trials which could translate into fewer visits during a clinical trial as serum markers are tracked (blood drawn locally). It also translates into additional targets for therapy development. And perhaps best of all, having serum biomarkers would enable trials to include young men of all ages, ambulatory or not.

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Sarepta Therapeutics Announces Plans to Submit Rolling NDA for Eteplirsen following Today’s Pre-NDA Meeting with the FDA

Sarepta Therapeutics Announces Plans to Submit Rolling NDA for Eteplirsen following Today’s Pre-NDA Meeting with the FDA | Duchenne Research | Scoop.it
ParentProjectMD's insight:

Great news! A huge step for Duchenne today as Sarepta announces plans to submit a rolling NDA for eteplirsen following today's pre-new drug application meeting with the FDA.

From PPMD President Pat Furlong:
"We are thrilled to see this amazing progress. It is a wonderful step forward for our community and we will do everything we can to encourage the FDA to rapidly review this submission. Tonight I will be sitting with Sarepta at a NORD gala. I will raise a toast to them, knowing that so many young men do not have the strength it takes to raise their arms. With all of them in mind, we thank Sarepta and wish them godspeed."

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EspeRare's Investigational Compound Rimeporide Receives European Orphan Drug Designation in Duchenne Muscular Dystrophy

GENEVA, May 4, 2015 /PRNewswire/ --

EspeRare today announced that the European Medicines Agency (EMA) has granted an...
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Summit Therapeutics Announces Phase 1B Modified Diet Clinical Trial Achieves Primary Objective In Duchenne Muscular Dystrophy

Summit Therapeutics Announces Phase 1B Modified Diet Clinical Trial Achieves Primary Objective In Duchenne Muscular Dystrophy | Duchenne Research | Scoop.it
Summit Therapeutics plc announced that its Phase 1b modified diet clinical trial of SMT C1100 for the treatment of Duchenne met its primary objective with half…
ParentProjectMD's insight:

Summit Therapeutics plc announced that its Phase 1b modified diet clinical trial of SMT C1100 for the treatment of Duchenne met its primary objective with half of the patients who received the higher dose of SMT C1100 achieving desired plasma levels while following specific dietary guidance.  Based on these results, Summit will advance SMT C1100 into a Phase 2 open-label clinical trial. Congratulations to our friends at Summit and we look forward to hearing more about next steps!

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BioMarin: Quarterly Update to the Duchenne Community (August 2015)

BioMarin: Quarterly Update to the Duchenne Community (August 2015) | Duchenne Research | Scoop.it
In an effort to provide regular communication on BioMarin's Duchenne muscular dystrophy programs to the Duchenne community, the company is circulating a quarte…
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Marathon Pharmaceuticals to Begin New Drug Application Process for Deflazacort as a Potential Treatment for Patients with Duchenne Muscular Dystrophy

Marathon Pharmaceuticals to Begin New Drug Application Process for Deflazacort as a Potential Treatment for Patients with Duchenne Muscular Dystrophy | Duchenne Research | Scoop.it
Marathon Pharmaceuticals, will begin the New Drug Application process for deflazacort as a potential treatment for patients with Duchenne muscular dystrophy
ParentProjectMD's insight:

Marathon Pharmaceuticals today announced that the company will begin the New Drug Application (NDA) process for deflazacort as a potential treatment for patients with Duchenne following a positive pre-NDA meeting with FDA. We are one step closer to approval of deflazacort in this country and we thank our partners at Marathon for their passion in the fight to end Duchenne!

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Fibrogen Receives FDA Clearance To Proceed With Clinical Study Of FG-3019 In Duchenne Muscular Dystrophy

FibroGen, Inc. (Nasdaq:FGEN), a research-based biopharmaceutical company, today announced that the U.S. Food and Drug Administration (FDA) has completed its review of the Company's investigational new drug (IND) application for the study of FG-3019 in patients with Duchenne muscular dystrophy (DMD), and clinical study may proceed. FG-3019 is a fully human monoclonal antibody that inhibits the activity of connective tissue growth factor (CTGF), a common mediator of fibrotic disease. FG-3019 is currently in Phase 2 clinical studies for idiopathic pulmonary fibrosis and pancreatic cancer.

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PPMD's 2015 Connect Conference Resources & Recordings - Part 1 (Thursday)

PPMD's 2015 Connect Conference Resources & Recordings - Part 1 (Thursday) | Duchenne Research | Scoop.it
The first batch of presentations from PPMD’s 21st Annual Connect Conference in Washington, D.C. are available for download. If you were unable to attend this…
ParentProjectMD's insight:

The first batch of presentations from PPMD’s 21st Annual Connect Conference in Washington, D.C. are available for download. If you were unable to attend this year’s conference or if you want to replay a presentation that you found particularly useful, we will be sharing all of the presentations over the next couple of weeks. 

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TREAT-NMD International Conference 2015 to be held in December in D.C.

TREAT-NMD Conference 2015
ParentProjectMD's insight:

The focus of this year's conference (their first in the U.S.) will be - Growing the drug pipeline for neuromuscular diseases: optimising resources for clinical development of new therapies

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FDA Accepts BioMarin's NDA for Drisapersen; Sarepta Completes NDA for Eteplirsen

FDA Accepts BioMarin's NDA for Drisapersen; Sarepta Completes NDA for Eteplirsen | Duchenne Research | Scoop.it
Big news in exon skipping therapy today! BioMarin announced this morning that the FDA has accepted their New Drug Application (NDA) for drisapersen and Sarepta…
ParentProjectMD's insight:

Big news in exon skipping therapy today! BioMarin announced this morning that the FDA has accepted their New Drug Application (NDA) for drisapersen and Sarepta announced that they have completed their NDA submission for eteplirsen.

We are such a fortunate community to have multiple companies working on multiple therapies to treat people with Duchenne. As always, PPMD will do everything we can to ensure that these therapies are reviewed as rapidly as possible. We are grateful to BioMarin and Sarepta for their commitment to Duchenne.

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PTC Therapeutics and BioMarin Pharmaceutical to Host DMD Industry Symposium

PTC Therapeutics, Inc. (NASDAQ: PTCT) and BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that they will host an Industry Update Symposium on Advances in Duchenne Muscular Dystrophy (DMD) and Biomarkers on Monday, June 22nd from 10 am – 1 pm ET at the Le Parker Meridien in New York City. Leading experts in the field will review the latest research on DMD, including natural history data, clinical biomarkers, and imaging assessments used in monitoring disease progression and response to therapy. An overview of state-of-the-art therapies in development for DMD will be discussed.

A live webcast of the event will be available on the Events and Presentations page under the investor relations section of PTC Therapeutics' and BioMarin's websites at www.ptcbio.com and at www.BMRN.com. A replay of the presentation will be archived. The presentation will be archived for 2 weeks following the presentation. It is recommended that users connect to the company website several minutes prior to the start of the webcast to ensure a timely connection.

 
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FDA Releases Draft Guidance for Duchenne

FDA Releases Draft Guidance for Duchenne | Duchenne Research | Scoop.it
Amazing things happen when this community works together, when our messages are consistent, and our goals are in sync. We work together, we change the landsca…
ParentProjectMD's insight:

BREAKING NEWS! Today the FDA has released their draft guidance on Duchenne. This is the first time that a patient community has proposed and drafted a guidance for FDA to use as a jumping off point for their own drug development guidance – an official map for how companies can move their Duchenne-focused innovation through the regulatory approval process.

 

Like you, PPMD is excited to dive in and find out what the FDA guidance says (and doesn’t say). And the process isn’t over. We, indeed anyone in the Duchenne community, can comment to the agency for the next 60 days. FDA will review those comments, make final tweaks, and then release a final guidance. And, in the meantime, we are told that both our draft guidance and theirs are available now to the review division to inform the review process. Our voice continues to matter.

 

Today we should celebrate. No more guessing games. We now have insight, directly from the source, about how FDA thinks about Duchenne. The guidance is here for us to read, think about, learn…then comment.

 

And together we will continue to change the landscape. Together, we will end Duchenne.

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Capricor Announces Receipt of FDA Clearance for the Phase I/II Study of CAP-1002 for the Treatment of Duchenne Muscular Dystrophy Related Cardiomyopathy

Capricor Announces Receipt of FDA Clearance for the Phase I/II Study of CAP-1002 for the Treatment of Duchenne Muscular Dystrophy Related Cardiomyopathy | Duchenne Research | Scoop.it
LOS ANGELES, June 8, 2015 (GLOBE NEWSWIRE) --
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Large-scale analysis of blood-based protein biomarkers for Duchenne muscular dystrophy published

Large-scale analysis of blood-based protein biomarkers for Duchenne muscular dystrophy published | Duchenne Research | Scoop.it
HACKENSACK, N.J. and BOULDER, Colo. , May 26, 2015 /PRNewswire-USNewswire/ -- The results of a...
ParentProjectMD's insight:

Breakthrough news! Today, SomaLogic and PPMD - together with several other Duchenne research groups - published a significant discovery that explains a technology allowing researchers to accurately measure the individual proteins in very small amounts of blood or other samples. By comparing patient and control samples, identification of critical differences in protein concentrations can be identified rapidly. This means that this data could be utilized as an outcome measure for clinical trials which could translate into fewer visits during a clinical trial as serum markers are tracked (blood drawn locally). It also translates into additional targets for therapy development. And perhaps best of all, having serum biomarkers would enable trials to include young men of all ages, ambulatory or not.

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PTC Therapeutics Launches STRIVE Grant Award Program

PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced it has launched STRIVE (Strategies to Realize Innovation, Vision and Empowerment), an awards program aimed at providing funds to patient advocacy organizations to develop unique and collaborative programs that will make meaningful contributions to the rare disease community by increasing awareness, diagnosis, education or fostering development of future patient advocates. The program, in its inaugural year, will focus on Duchenne muscular dystrophy (DMD).

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