Duchenne Nation Research News
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Duchenne Nation Research News
Newswire articles about Duchenne muscular dystrophy research, treatment and education.
Curated by Deb Robins
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FDA decision on eteplirsen delayed - Center for Duchenne Muscular Dystrophy at UCLA 

FDA decision on eteplirsen delayed - Center for Duchenne Muscular Dystrophy at UCLA  | Duchenne Nation Research News | Scoop.it

Dear CDMD Supporters,

The FDA delayed decision on the approval for eteplirsen.

We will keep updating you throughout the process.

Thanks for all your support and interest.

Deb Robins's insight:
The delay is disappointing. CDMD stakeholders feel the FDA are conflicted but they are hopeful that the delay is not long and the decision is for accelerated approval. 
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Sarepta AdCom Should Set The Record Straight

Sarepta AdCom Should Set The Record Straight | Duchenne Nation Research News | Scoop.it
Before the FDA renders its decision on Sarepta’s eteplirsen on May 26, I’d like to ask the narrow majority on the advisory committee who voted “no” on the question of accelerated approval whether they would still vote the same way after some reflection. The lives of thousands of boys with Duchenne Muscular Dystrophy (DMD) will be affected by this decision and many believe that it was the confusing wording of the question that stacked the deck against them. The least we can do is set the record straight and reassure them that the panelists had good reasons for their votes.
Deb Robins's insight:
Quote: “It is possible for a medication to have an effect without knowing why. We have tylenol. We have heparin. We don’t know how they work. Doesn’t mean that we should throw them out.” The preponderance of the evidence standard of proof leaves some room for doubt so you don’t have to throw out eteplirsen because we don’t yet understand how it works."
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PTC Stock Could Be A Better Buy -- The Motley Fool

PTC Stock Could Be A Better Buy --  The Motley Fool | Duchenne Nation Research News | Scoop.it
PTC Therapeutics is already generating revenue from its Duchenne muscular dystrophy drug, and its financial picture looks stronger than Sarepta Therapeutics'.
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Parent Project Muscular Dystrophy Convenes Bone Health and Osteoporosis Workshop

Parent Project Muscular Dystrophy Convenes Bone Health and Osteoporosis Workshop | Duchenne Nation Research News | Scoop.it

Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne) will convene a Bone Health and Osteoporosis Workshop spanning translational research and clinical care, May 12-13. PPMD has pulled together more than 50 international experts in both pediatric and Duchenne bone health surveillance/monitoring/treatment to discuss a wide range of topics.

Deb Robins's insight:
Like any good builder, PPMD, has built strong foundations, extended the house in new directions but reviews any new cracks, seeking to fill them in. 
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Newborn screening for Duchenne back in the spotlight - UK

Newborn screening for Duchenne back in the spotlight - UK | Duchenne Nation Research News | Scoop.it
A recent Muscular Dystrophy UK survey on newborn screening and All Party Parliamentary Group for Muscular Dystrophy report found that: even without a treatment currently available, 82 percent of parents interviewed were in favour of screening for Duchenne at birth early diagnosis of Duchenne allows parents to anticipate health, housing, educational and emotional needs of their child and to manage the onset of symptoms through cardiac care, physiotherapy and steroid treatments. screening could reduce the number of parents who unknowingly go on to have two or even three affected children.
Deb Robins's insight:
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Study find Deflazacort, a Potential DMD Drug, Superior to Prednisone in Functional Tests, Weight Gain 

Study find Deflazacort, a Potential DMD Drug, Superior to Prednisone in Functional Tests, Weight Gain  | Duchenne Nation Research News | Scoop.it

Read about Marathon Pharmaceuticals's positive findings from a Phase 3 trial comparing deflazacort to prednisone and placebo in boys with Duchenne MD.

Deb Robins's insight:
There's nothing "potential" about Deflazacort and it's been long known to be efficacious. As for superiority over Prednisone, that has been touted also for a very long time. More importantly, it is about choice of drug if one is unsuitable for one reason or another. 
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The FDA Needs A Conditional Approval System

The FDA Needs A Conditional Approval System | Duchenne Nation Research News | Scoop.it

BY JOSEPH GULFO - What is disappointing to DMD patients and their families, and to millions of other Americans, is that the FDA does not have a real conditional approval system. Perhaps, the eteplirsen debate will finally impel the FDA or Congress to establish such as system, akin to the EMA. Dr. Gulfo is also author of “Innovation Breakdown: How the FDA and Wall Street Cripple Medical Advances” (Post Hill Press) and the former CEO of Mela Sciences.  Gulfo is author of: Innovation Breakdown: How the FDA and Wall Street Cripple Medical Advances

Deb Robins's insight:
We have one world environment and each of us responsible for consuming and emitting an unfathomable number of chemicals. The EMA has a true conditional approval system whereby drugs with some indication of benefit can be definitively tested over a series of confirmatory studies. It should be a universal concept.
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Corticosteroids for the treatment of DMD - A Cochrane Meta-Analysis

Corticosteroids for the treatment of DMD - A Cochrane Meta-Analysis | Duchenne Nation Research News | Scoop.it

AUTHORS' CONCLUSIONS: Moderate quality evidence from RCTs indicates that corticosteroid therapy in DMD improves muscle strength and function in the short term (twelve months), and strength up to two years. On the basis of the evidence available for strength and function outcomes, our confidence in the effect estimate for the efficacy of a 0.75 mg/kg/day dose of prednisone or above is fairly secure. There is no evidence other than from non-randomised trials to establish the effect of corticosteroids on prolongation of walking. In the short term, adverse effects were significantly more common with corticosteroids than placebo, but not clinically severe. A weekend-only prednisone regimen is as effective as daily prednisone in the short term (12 months), according to low to moderate quality evidence from a single trial, with no clear difference in BMI (low quality evidence). Very low quality evidence indicates that deflazacort causes less weight gain than prednisone after a year's treatment. We cannot evaluate long-term benefits and hazards of corticosteroid treatment or intermittent regimens from published RCTs. Non-randomised studies support the conclusions of functional benefits, but also identify clinically significant adverse effects of long-term treatment, and a possible divergence of efficacy in daily and weekend-only regimens in the longer term. These benefits and adverse effects have implications for future research and clinical practice.Cochrane Database Syst Rev. 2016 May 5;5:CD003725. [Epub ahead of print] REVIEW

Deb Robins's insight:
Even given variation, this meta-analysis would seem to underplay the effect of Corticosteroids - particularly if commenced early in childhood. 
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Coalition Duchenne Says FDA Panel Missed 'Complexity' of DMD 

Coalition Duchenne Says FDA Panel Missed 'Complexity' of DMD  | Duchenne Nation Research News | Scoop.it
Read the reaction of Coalition Duchenne's founder to the recent FDA panel vote not to grant accelerated approval to eteplirsen as a DMD treatment. 
Deb Robins's insight:
I have no answers only questions.
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Concer over muscular dystrophy drug decision

Concer over muscular dystrophy drug decision | Duchenne Nation Research News | Scoop.it

... the National Centre for Pharmacoeconomics (NCPE), which facilitates healthcare decisions on the reimbursement of treatments using clinical and scientific evidence, has decided not to recommend Translarna for reimbursement here. According to John Bennett of MDI, ‘it is only fair and reasonable' that Irish children should have the same opportunities as children elsewhere in Europe. "It is our information that this new treatment would only apply in Ireland to a very small number of patients - in the low single digits. However, for those young people it would make a huge difference in slowing progression of the condition and in keeping them on their feet for longer.

Deb Robins's insight:
The annual cost for a ten-year old of average weight is €411,136 according to the bean counters. 
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Santhera Updates on Regulatory Filings for Raxone® (idebenone) in DMD

Santhera Updates on Regulatory Filings for Raxone® (idebenone) in DMD | Duchenne Nation Research News | Scoop.it
Santhera Pharmaceuticals (SIX: SANN) announces that it has submitted comprehensive briefing material and a meeting request to the FDA to discuss the filing of a New Drug Application (NDA) for Raxone® (idebenone) for the treatment of DMD patients not taking concomitant glucocorticoids. A Marketing Authorization Application for DMD in Europe is also expected to be submitted in coming weeks. The Company also reports that, as part of its strategy to expand the Raxone label, a new phase III study (SIDEROS) in DMD patients using glucocorticoids will start enrolling patients in coming weeks.
Deb Robins's insight:
Approval of this compound should see us breathing easier!
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Duchenne Expert Testemonies at Eteplirsen AdCom April 25th

Duchenne Expert Testemonies at Eteplirsen AdCom April 25th | Duchenne Nation Research News | Scoop.it
On Monday, April 25th, we made Duchenne history by changing forever the way patient input is considered by the FDA and the drug development world. Rare disease patients and advocates around the nation will benefit from your groundbreaking actions. While Duchenne patients and their families know know first hand that eteplirsen works and is safe; listen to the testimonies below from world renowned Duchenne experts.
Deb Robins's insight:
The scales tip more and more towards individual case notes as opposed flawed data. 
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Pat Furlong's spoken Eteplirsen Testmony

Pat Furlong's spoken Eteplirsen Testmony | Duchenne Nation Research News | Scoop.it
PPMD Founding President & CEO, Pat Furlong speaking during today's Open Public Hearing for eteplirsen. #MakeDuchenneHistory
Deb Robins's insight:
May be found on PPMD's FB timeline, April 26. 2016.
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UCLA's Stem Cell Gene Therapy Could Treat DMD 

UCLA's Stem Cell Gene Therapy Could Treat DMD  | Duchenne Nation Research News | Scoop.it

Researchers at the UCLA Center for Duchenne Muscular Dystrophy and Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research have been working on a new approach that can potentially be used to treat Duchenne muscular dystrophy (DMD). “This method is likely 10 years away from being tested in people,” UCLA Professor of neurology and co-director of the Center for Duchenne Muscular Dystrophy at UCLA, Melissa Spencer, said in a news release. “It is important that we take all the necessary steps to maximize safety while quickly bringing a therapeutic treatment to patients in clinical trials.” The promising approach explores a technology called CRISPR/Cas9 (Clustered Regularly Interspaced Short Palindromic Repeats) to correct genetic mutations that trigger the disease. 

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The FDA should approve the first disease-modifying treatment for DMD

The FDA should approve the first disease-modifying treatment for DMD | Duchenne Nation Research News | Scoop.it
Deb Robins's insight:
Hope floats on. 
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What's the Fuss?

What's the Fuss? | Duchenne Nation Research News | Scoop.it
What's the Fuss? What's the Fuss about medical research? What's the Fuss about the Medical Research Future Fund?
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Wave Life Sciences touts more effective Duchenne drug

Wave Life Sciences touts more effective Duchenne drug | Duchenne Nation Research News | Scoop.it

“We recognize the acute need of the Duchenne community for therapeutic options to address this devastating disease, and also appreciate regulators’ requirements for strong, well-validated, scientific evidence. Our goal is to fulfill both of these needs," said Paul Bolno, CEO of Wave.BioPharma

Deb Robins's insight:
I don't quite understand why this "given"  from a Pharma CEO seems so refreshing. 
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Capricor Therapeutics Update and First Quarter 2016 Financial Results

Capricor Therapeutics Update and First Quarter 2016 Financial Results | Duchenne Nation Research News | Scoop.it

Upcoming Milestones: Complete enrollment in the HOPE-Duchenne and ALLSTAR trials in the third quarter of 2016 Report 12-month results from the DYNAMIC trial in advanced heart failure in the third quarter of 2016 Report six-month top-line data from HOPE-Duchenne in the first quarter of 2017 Six-month top-line data from ALLSTAR expected in the first quarter of 2017LOS ANGELES, May 12, 2016 /PRNewswire/ -- Capricor Therapeutics Provides Corporate Update and Reports First Quarter 2016 Financial Results

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Wave Planning to Start Clinical Testing of its Exon 51-Skipping Therapy for DMD in 2017

Wave Planning to Start Clinical Testing of its Exon 51-Skipping Therapy for DMD in 2017 | Duchenne Nation Research News | Scoop.it

The company will present its data at the Parent Project Muscular Dystrophy’s (PPMD) Annual Connect Conference, taking place June 26–29 in Orlando, Florida. “We are excited whenever a new company enters the Duchenne space,” said Pat Furlong, PPMD’s founding president and chief executive officer. “Individuals with Duchenne need and deserve options, and we are hoping this will become another tool in our arsenal.”Read about Wave Life Sciences plan to begin clinical testing of its Exon 51-skipping therapy for patients with Duchenne muscular dystrophy in 2017.

Deb Robins's insight:
More about this promising new technology if not exactly a new approach.  Hope springs...
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Pat Furlong On Eteplirsen: Disappointing But A Big Step Forward

Pat Furlong On Eteplirsen: Disappointing But A Big Step Forward | Duchenne Nation Research News | Scoop.it

Furlong also believes starting DMD-diagnosed boys on the drug earlier could have a significant impact on their lives. With new advances in screening, a child might be diagnosed with the disease at age two or three and immediately start treatments. That would result in a far different scenario than waiting until the patient is seven years old and has lost up to 50 percent of his muscle. “By seven years, the loss of dystrophin and the physiological cascade that occurs after that loss of dystrophin is difficult to reverse,” notes Furlong. “Even if eteplirsen restores only small amounts of dystrophin, it would begin to impact that physiological cascade, and that is a good start.”A recent FDA advisory committee hearing on the Duchenne muscular dystrophy drug eteplirsen resulted in a recommendation against approval. Pat Furlon

Deb Robins's insight:
Very interesting and optimistic interview in the Clinical Leader with the worlds most knowledgeable patient-medico, Pat Furlong. An open label confirmatory study would indeed test the benefits of early intervention we have come to know in all aspects of treatment and care. 
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Cholesterol Drug, Simvastatin as Effective as Current Gene Therapy in DMD Mice

Cholesterol Drug, Simvastatin as Effective as Current Gene Therapy in DMD Mice | Duchenne Nation Research News | Scoop.it
Read about how the cholesterol-lowering drug simvastatin reverses fibrosis and improves muscle strength in a mouse model of Duchenne muscular dystrophy.
Deb Robins's insight:
We know that first generation gene therapy currently isn't a high standard to aspire to, but remember also that mice is mice and men is men - and they match up only 5% of the time.
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NICE gives PTC, NHS more time to agree Duchenne drug price 

NICE gives PTC, NHS more time to agree Duchenne drug price  | Duchenne Nation Research News | Scoop.it
NICE has given PTC Therapeutics extra time to thrash out a cost-effective price plan with NHS England before it will allow a final yes for funding through the country’s state-run healthcare system.
Deb Robins's insight:
NICE estimates the average cost (as it is dosed according to weight) to be around £220,256.  Much lower than the Irish estimate.
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CDMRP Duchenne MD Research Program 2016

Defense Health Program Department of Defense Duchenne Muscular Dystrophy Research Program Funding Opportunities for Fiscal Year 2016 The Fiscal Year 2016 (FY16) Defense Appropriations Act provides $3.2 million (M) to the Department of Defense Duchenne Muscular Dystrophy Research Program (DMDRP) to support innovative, high-impact Duchenne muscular dystrophy research. As directed by the Office of the Assistant Secretary of Defense for Health Affairs, the Defense Health Agency, Research, Development, and Acquisition (DHA RDA) Directorate manages and executes the Defense Health Program (DHP) Research, Development, Test, and Evaluation (RDT&E) appropriation. The managing agent for the anticipated Program Announcements/Funding Opportunities is the Congressionally Directed Medical Research Programs (CDMRP). The DMDRP is providing the information in this pre-announcement to allow investigators time to plan and develop applications. FY16 DMDRP Program Announcements and General Application Instructions for the following award mechanisms are anticipated to be posted on the Grants.gov website in late May 2016. Pre-application and application deadlines will be available when the Program Announcements are released. This pre-announcement should not be construed as an obligation by the government.
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Benjamin Dupree on Eteplirsen

"Living with duchenne I don't know how that's even possible."
Deb Robins's insight:
From objectivity, critical thinking, experience and internal conflict arises THE most insightful argument to date. 
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DON’T SKIP DMD PATIENTS

DON’T SKIP DMD PATIENTS | Duchenne Nation Research News | Scoop.it
The skimpy NDA for eteplirsen to treat Duchenne muscular dystrophy has FDA wedged between the rock of inconclusive data and the hard place of a well-informed patient community that understands the limitations of the data and still demands access to the compound. There are very good arguments for rejecting eteplirsen, but they are outweighed by the possibility that the compound might help and is very unlikely to harm boys who have no other hope.
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