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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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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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Parent Project Muscular Dystrophy and Duchenne Parent Project (Netherlands) to Host International Robotics Workshop on April 27

Parent Project Muscular Dystrophy and Duchenne Parent Project (Netherlands) to Host International Robotics Workshop on April 27 | Duchenne Research | Scoop.it
HACKENSACK, N.J., April 27, 2015 /PRNewswire-USNewswire/ -- Parent Project Muscular Dystrophy (PPMD), a...
ParentProjectMD's insight:

Today in London, PPMD is joining Duchenne stakeholders from around the world in a special one-day meeting to discuss robotic technology and the potential it holds for our community. PPMD – together with UPPMD – has gathered the best and brightest robotics experts and engineers who are working to apply their innovations to Duchenne. Today we are discussing the variety of technologies currently in development – technologies that inspired PPMD to launch our Robotics Initiative earlier this spring. A program that, to date, has raised $110,000! ‪#‎letsinnovate‬

Support PPMD's Robotics Initiative: www.parentprojectmd.org/letsinnovate

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PhaseBio Announces Promising Pre-Clinical Results for PB1046 in Models of Duchenne Muscular Dystrophy

MALVERN, Pa., April 22, 2015 (GLOBE NEWSWIRE) -- PhaseBio Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company and leader in the field of biopolymer-based drugs focused on developing treatments for metabolic and specialty cardiopulmonary disorders, announced that positive pre-clinical data on its compound PB1046 in Duchenne muscular dystrophy (DMD) models will be presented today in the emerging science poster session at the 67
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Santhera Releases Positive Data from Phase III Clinical Trial of Idebenone (Raxone®/Catena®) in Duchenne

Santhera Releases Positive Data from Phase III Clinical Trial of Idebenone (Raxone®/Catena®) in Duchenne | Duchenne Research | Scoop.it
Today, Santhera has published the results in the highly prestigious journal, The Lancet, from their successful clinical trial of Raxone®/Catena® . They will pr…
ParentProjectMD's insight:

Today, Santhera has published the results from their successful clinical trial of idebenone (Raxone®/Catena®) in Duchenne. First, idebenone was safe and well tolerated. Importantly, treatment of study subjects, age 10-18, for one year with Raxone®/Catena® reduced the drop in an important respiratory measurement. The decline in peak expiratory flow was decreased by 66% compared to placebo. In addition, Raxone®/Catena® had a positive effect on several other respiratory function measures.


This is an important and certainly not a modest effect on respiratory function and may well have a substantial effect on quality of life for Duchenne. Santhera has stated their intent to file for regulatory approval in the U.S. and Europe very soon. We very much look forward to the approval of Raxone®/Catena® and to the potential use of the drug in a combination therapy approach to Duchenne.

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First Duchenne Muscular Dystrophy Patient Treated in Follistatin Gene Therapy Trial

First Duchenne Muscular Dystrophy Patient Treated in Follistatin Gene Therapy Trial | Duchenne Research | Scoop.it
CLEVELAND and COLUMBUS, Ohio, April 13, 2015 /PRNewswire-USNewswire/ -- Milo Biotechnology announced the first...
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Sarepta Update

Sarepta Update | Duchenne Research | Scoop.it
Earlier this month Sarepta announced a change in leadership, with CMO Ed Kaye taking over as interim CEO.
Following that announcement the team at Sarepta orga…
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Earlier this month Sarepta announced a change in leadership, with CMO Ed Kaye taking over as interim CEO. Following that announcement the team at Sarepta organized a call with Duchenne patient organizations and foundations to update them on the transition. Here is a short update from the team at Sarepta regarding their ongoing Duchenne program.

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Sarepta Therapeutics Appoints Edward Kaye, M.D., as Interim CEO; Company on Track with Clinical and Regulatory Plans for Investigational Duchenne Muscular Dystrophy Drugs

Sarepta Therapeutics Appoints Edward Kaye, M.D., as Interim CEO; Company on Track with Clinical and Regulatory Plans for Investigational Duchenne Muscular Dystrophy Drugs | Duchenne Research | Scoop.it

Sarepta Therapeutics, Inc. today announced the appointment of Edward Kaye, M.D., the company’s Chief Medical Officer (CMO), as interim Chief Executive Officer (CEO) effective immediately. He replaces Christopher Garabedian, who resigned as President and Chief Executive Officer and as a member of the Board, also effective immediately. Dr. Kaye, who will continue in a dual capacity as CEO and CMO while the company conducts a search for a new full-time CEO, will focus his efforts on heading the regulatory and clinical process for the company’s lead product candidate, eteplirsen, and follow on “exon” drug candidates for Duchenne Muscular Dystrophy (DMD).

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Dr. Jerry Mendell Discusses Phase 1/2a Follistatin Gene Therapy Trial for Becker Muscular Dystrophy :: March 2015

Dr. Jerry Mendell Discusses Phase 1/2a Follistatin Gene Therapy Trial for Becker Muscular Dystrophy :: March 2015 | Duchenne Research | Scoop.it
Dr. Jerry Mendell Discusses Phase 1/2a Follistatin Gene Therapy Trial for Becker Muscular Dystrophy in
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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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BioMarin Completes Rolling NDA Submission to FDA for Drisapersen for Treatment of Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping (NASDAQ:BMRN)

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The Duchenne community should celebrate. Today, Biomarin submitted the rolling NDA on drisapersen. This means we now have a second NDA submitted for Duchenne. Santhera is likely to follow soon. By first quarter 2016, we may see 3 approved drugs with more to follow. This is exactly what we have been working for, hoping for.

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Capricor Granted FDA Orphan Drug Designation for Allogeneic Cardiosphere-Derived Cells for the Treatment of Duchenne Muscular Dystrophy

Capricor Granted FDA Orphan Drug Designation for Allogeneic Cardiosphere-Derived Cells for the Treatment of Duchenne Muscular Dystrophy | Duchenne Research | Scoop.it

Capricor Therapeutics, Inc. (Nasdaq:CAPR), a biotechnology company focused on developing novel therapeutics for the treatment of cardiovascular diseases, today announced that it has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA) for its cell therapeutic product candidate, CAP-1002, for the treatment of cardiomyopathy associated with Duchenne muscular dystrophy (DMD).

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Duchenne Muscular Dystrophy market to hit $990m by 2019

The global treatment market for Duchenne Muscular Dystrophy will expand in value at a CAGR of 160.5% to $990 million by 2019, says GlobalData.
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BioMarin Update

BioMarin Update | Duchenne Research | Scoop.it
BioMarin has provided the following update on redosing of drisapersan for a new clinical trial. Ambulation will not be an inclusion or exclusion criterion for…
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BioMarin has provided the following update on redosing of drisapersen for a new clinical trial. Ambulation will not be an inclusion or exclusion criterion for enrollment, but boys will need to have been previously dosed as part of a clinical trial.

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Santhera receives FDA Fast Track Designation for Raxone®/Catena® (idebenone) for theTreatment of Duchenne Muscular Dystrophy

Santhera receives FDA Fast Track Designation for Raxone®/Catena® (idebenone) for theTreatment of Duchenne Muscular Dystrophy | Duchenne Research | Scoop.it
ParentProjectMD's insight:

The U.S. Food and Drug Administration (FDA) has granted Fast Track designation for Santhera's Raxone®/Catena® (idebenone) for the treatment of Duchenne muscular dystrophy. FDA's Fast Track program facilitates the development and review of important drugs intended to treat serious conditions and fill an unmet medical need for the purpose of getting them to the patient earlier. Santhera previously announced that the Phase III trial (DELOS) in Duchenne met its primary endpoint and demonstrated that Raxone/Catena delayed the loss of respiratory function.

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Recapping the FDA-NIH Dystrophin Methodology Workshop

Recapping the FDA-NIH Dystrophin Methodology Workshop | Duchenne Research | Scoop.it
I always felt a strong connection to the Duchenne community while working at the NIH. As you know, that connection and the incredible work of organizations li…
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PPMD CEO, Dr. John Porter, recaps the FDA and NIH dystrophin methodology workshop. This was an important moment for the Duchenne community and PPMD was honored to be a part of it. Thank you to all the families who came to the meeting.

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