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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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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...
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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…
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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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PPMD Participating in FDA-NIH Dystrophin Methodology Meeting on Friday, March 20

PPMD Participating in FDA-NIH Dystrophin Methodology Meeting on Friday, March 20 | Duchenne Research | Scoop.it
John Porter and I have been invited to participate in the FDA-NIH dystrophin methodology meeting on Friday, March 20. While we said yes, we realize this is a…
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PPMD is grateful that the FDA and NIH are willing to discuss dystrophin and have opened their doors to include our community leaders. If you would like to attend Friday’s public scientific workshop, see details here: http://1.usa.gov/1GYNSpV. If you cannot attend but would like to watch a live stream of the meeting, view this link Friday morning to attend virtually: http://1.usa.gov/1GYNSpV.
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Register for Webinar: Understanding the Duchenne Muscular Dystrophy (DMD) Market Landscape: A Closer Look at the Current Trends and Future Market Dynamics

Register for Webinar: Understanding the Duchenne Muscular Dystrophy (DMD) Market Landscape: A Closer Look at the Current Trends and Future Market Dynamics | Duchenne Research | Scoop.it
In this webinar GlobalData’s Analyst covering Immunology, Dr. Nikhilesh Sanyal, will share insights pulled from GlobalData’s Opportunity Analyzer: Duchenne Muscular Dystrophy – Opportunity Analysis and Forecast to 2019 report. Dr. Sanyal will outline and review the current DMD treatment landscape, discuss unmet needs and opportunities, and present GlobalData’s comprehensive analysis of DMD pipeline drugs. Dr. Toli Koutsokeras, GlobalData’s Senior Analyst covering Immunology, will moderate a Q&A session following the presentation.

Duchenne muscular dystrophy (DMD) represents a truly nascent market with a very strong demand for effective therapies from the patient community and clinicians. DMD is a rare and fatal genetic disorder characterized by progressive muscle degeneration, loss of ambulation, and death by the late 20s. The current standard of treatment involves generic corticosteroids, such as prednisone and deflazacort, which are used to marginally prolong muscle function and are not disease-modifying therapies.

The major obstacles that hinder the development of a clinically effective therapeutic for DMD are the multitude of DMD mutations and non-uniform progression of the disease. The two largest groups of mutations which can be targeted by a single therapeutic molecule are mutations clustered around exon-51 (approximately 13% of all DMD cases) and nonsense mutations (approximately 12.8% of all DMD cases) in the DMD gene. Hence, drug developers have to strike a delicate balance of targeting specific DMD patient populations while demonstrating sufficient clinical efficacy.

Join this webinar to increase your knowledge of the current state of the DMD market and what can be expected in this exciting therapeutic area in the future.
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Principal Investigator to Present Phase III Data on Idebenone in Duchenne Muscular Dystrophy (DMD) at the Annual Meeting of the American Academy of Neurology (AAN) - News Press Release | PharmiWeb.com

Principal Investigator to Present Phase III Data on Idebenone in Duchenne Muscular Dystrophy (DMD) at the Annual Meeting of the American Academy of Neurology (AAN) - News Press Release | PharmiWeb.com | Duchenne Research | Scoop.it
Liestal Switzerland March 11 2015- Santhera Pharmaceuticals (SIX: SANN) announces that Gunnar M. Buyse PhD MDProfessor of Child Neurology at the University Hospitals Leuven (Belgium) and principal investigator will present outcome data of the Phase I
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Dystrophin 101 Webinar (March 2015) - YouTube

Dystrophin 101: Everything You Always Wanted to Know About the Duchenne Protein (and were not afraid to ask) This webinar reviewed the basic biology of dystr...
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The recording of our Dystrophin 101 webinar is now available. Learn more about the basic biology of dystrophin, how dystrophin is measured in both preclinical studies and clinical trials, and the need for and role that dystrophin analysis play in clinical trials of novel agents designed to exert their therapeutic effects through an increase in dystrophin levels. Our speakers also discussed what is required in order to have dystrophin considered as a surrogate.

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Sarepta Therapeutics Announces Fourth Quarter and Full-Year 2014 Financial Results and Recent Corporate Developments

Sarepta Therapeutics Announces Fourth Quarter and Full-Year 2014 Financial Results and Recent Corporate Developments | Duchenne Research | Scoop.it

- NDA submission for eteplirsen planned for mid-year 2015 -

- Plan to discuss new data and NDA submission with FDA in 2nd Quarter –

- Cash and Other Investments of $211 Million at Year End 2014 -

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Upcoming Webinar - Dystrophin 101: Everything You Always Wanted to Know About the Duchenne Protein (And Were Not Afraid to Ask)

Upcoming Webinar - Dystrophin 101: Everything You Always Wanted to Know About the Duchenne Protein (And Were Not Afraid to Ask) | Duchenne Research | Scoop.it

March 3, 2015 from 1pm to 2pm ET
This webinar will review the basic biology of dystrophin (the gene, structure-function of the protein, its role in the dystrop…

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March 3, 2015 from 1pm to 2pm ET

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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
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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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Nicox receives Orphan Drug Designation from FDA for naproxcinod in Duchenne Muscular Dystrophy

SOPHIA ANTIPOLIS, France, March 19, 2015 (GLOBE NEWSWIRE) --
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What is Epicatechin?

What is Epicatechin? | Duchenne Research | Scoop.it
Epicatechin is a flavonoid found in dark chocolate harvested from the cacao tree. What’s that got to do with Duchenne? Epicatechin is one of dozens of flavono…
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Epicatechin. It’s a word that is starting to pop up more and more in the Duchenne community. But was is it? PPMD helps explain this exciting potential therapy now in the early stages of development.

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Idera Pharmaceuticals Reports Fourth Quarter and Year End 2014 Financial Results and Provides Corporate Update

Idera Pharmaceuticals Reports Fourth Quarter and Year End 2014 Financial Results and Provides Corporate Update | Duchenne Research | Scoop.it
CAMBRIDGE, Mass. and EXTON, Pa., March 12, 2015 (GLOBE NEWSWIRE) -- Idera Pharmaceuticals, Inc. (Nasdaq:IDRA), a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of novel therapeutics for oncology and rare diseases, today reported its financial and operational results for the fourth quarter and year ended December 31, 2014.
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BioMarin Update on Duchenne Muscular Dystrophy program (March 2015)

BioMarin Update on Duchenne Muscular Dystrophy program (March 2015) | Duchenne Research | Scoop.it
United Parent Projects Muscular Dystrophy (UPPMD) requested an update from Biomarin seeking answers to specific questions around next steps in terms of re-dosi…
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United Parent Projects Muscular Dystrophy (UPPMD) requested an update from Biomarin seeking answers to specific questions around next steps in terms of re-dosing, NDA submission for drisapersen in the US, the development program for additional exons, and natural history study. Read the update here.

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PTC Announces Translarna™ Access Program In Duchenne Muscular Dystrophy for Siblings of Patients Participating in PTC Clinical Trials (NASDAQ:PTCT)

PTC Therapeutics, Inc., today announced that it will make Translarna available for siblings of patients in certain open label PTC clinical trials for nonsense mutation Duchenne muscular dystrophy (nmDMD). Translarna will be made available to these siblings in advance of commercial availability in their regions, after a treating physician and the patient or guardian make a clinical decision to use Translarna, and consistent with any other applicable regulatory requirements.

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PTC Therapeutics today announced that it will make Translarna available for siblings of patients in certain open label PTC clinical trials for nonsense mutation Duchenne in advance of commercial availability in their regions.

“Families of children with Duchenne have wanted companies to be responsive in the case of siblings. With this program, PTC, once again, demonstrates itself as a champion for the Duchenne community.”

-- Pat Furlong, Founding President of PPMD

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Parent Project Muscular Dystrophy & Foundation to Eradicate Duchenne Award $1 Million Grant to ReveraGen BioPharma

Parent Project Muscular Dystrophy & Foundation to Eradicate Duchenne Award $1 Million Grant to ReveraGen BioPharma | Duchenne Research | Scoop.it
Parent Project Muscular Dystrophy (PPMD) is excited to join forces with Foundation to Eradicate Duchenne (FED) to award ReveraGen BioPharma (ReveraGen) a $1…
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