Duchenne Research
Follow
Find
67.1K views | +8 today
 
Scooped by ParentProjectMD
onto Duchenne Research
Scoop.it!

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.     

more...
Filippo Buccella's curator insight, May 23, 2014 10:28 AM

Great Great day! Agree!

From around the web

Duchenne Research
Your new post is loading...
Your new post is loading...
Scooped by ParentProjectMD
Scoop.it!

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.
more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

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…
ParentProjectMD's insight:

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.

more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

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.

more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

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…
ParentProjectMD's insight:

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.

more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

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

SOPHIA ANTIPOLIS, France, March 19, 2015 (GLOBE NEWSWIRE) --
more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

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…
ParentProjectMD's insight:

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.

more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

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.
more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

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…
ParentProjectMD's insight:

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.

more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

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.

ParentProjectMD's insight:

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

more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

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…
more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

PPMD Awards University of Missouri $50,000 Exploratory Grant to Explore SERCA2A in Duchenne Dog Study

PPMD Awards University of Missouri $50,000 Exploratory Grant to Explore SERCA2A in Duchenne Dog Study | Duchenne Research | Scoop.it
PPMD has awarded a $50,000 Exploratory Grant to University of Missouri to test SERCA2A gene therapy as a potential treatment for dogs with Duchenne. Dr. Dongs…
more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

Parent Project Muscular Dystrophy Awards UCLA $50,000 Exploratory Grant for Evaluation of Exon Skipping Enhancers in Duchenne

Parent Project Muscular Dystrophy Awards UCLA $50,000 Exploratory Grant for Evaluation of Exon Skipping Enhancers in Duchenne | Duchenne Research | Scoop.it
PPMD announced today that they will award Dr. M. Carrie Miceli and her team at UCLA’s David Geffen School of Medicine and College of Letters and Science, a $5…
more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

Duchenne muscular dystrophy drug could get OK for U.S. sales in 2016

Duchenne muscular dystrophy drug could get OK for U.S. sales in 2016 | Duchenne Research | Scoop.it
A Northbrook company said it has won fast-track status for approval of a drug that could become the first in the United States to treat Duchenne muscular dystrophy , a fatal muscle disease found most often in boys.
more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

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...
more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

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…
ParentProjectMD's insight:

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.

more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

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).

more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

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
more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

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…
ParentProjectMD's insight:
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.
more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

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.
more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

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
more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

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...
ParentProjectMD's insight:

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.

more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

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 -

more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

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…

ParentProjectMD's insight:

March 3, 2015 from 1pm to 2pm ET

more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

Summit shares climb as unveils US listing plans

Summit shares climb as unveils US listing plans | Duchenne Research | Scoop.it
Shares in drug developer Summit jumped more than 10% on Monday as it told investors it continues to make progress on a number of fronts and unveiled plans to list on the NASDAQ exchange in the US.
more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

Rare feat: BioMarin rewrites playbook for small-market drug approvals

Rare feat: BioMarin rewrites playbook for small-market drug approvals | Duchenne Research | Scoop.it
BioMarin Pharmaceutical looks to Europe to save time and money in developing rare-disease drugs.
more...
No comment yet.