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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Effects of Mechanical InsufflationExsufflation on the Breathing Pattern in Stable Subjects With Duchenne Muscular Dystrophy: A Step in a Wrong Direction

Effects of Mechanical InsufflationExsufflation on the Breathing Pattern in Stable Subjects With Duchenne Muscular Dystrophy: A Step in a Wrong Direction | Duchenne Nation Research News | Scoop.it
 Many clinicians are struggling to use optimal settings when using MI-E within the goals for which this device was designed.We believe that MI-E studies must focus on assisting cough rather than improving breathing pattern and lung mechanics.
Deb Robins's insight:
Toussaint et al make some good points here about using the right respiratory tool for the intended job.
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Gene Therapy & Duchenne Muscular Dystrophy

Gene therapy for Duchenne muscular dystrophy is centered on the goal of successfully introducing a smaller, but efficient version of dystrophin into the muscle cell.
Deb Robins's insight:
The latest sketch animated explainer from PPMD helps visual thinkers.
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Sarepta's gene therapy program for Duchenne resumes after clinical trial hold lifted

Sarepta's gene therapy program for Duchenne resumes after clinical trial hold lifted | Duchenne Nation Research News | Scoop.it
Sarepta’s early-stage clinical trial for a gene therapy for Duchenne muscular dystrophy is back on schedule, the company announced.
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Duchenne Parent Project Sept 2018 Care Conference

Duchenne Parent Project Sept 2018 Care Conference | Duchenne Nation Research News | Scoop.it

Catch all the international presentations from the #WDAD18 Standards of Care Conference in Amsterdam on the DPP youtube channel.

Deb Robins's insight:
Very seamless live stream. Thank you to DPP.
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Standards of Care do Matter  #WDAD18

Standards of Care do Matter  #WDAD18 | Duchenne Nation Research News | Scoop.it
To celebrate the theme of #WDAD18, Doug designed a #1 Cardiologist mug with the WDAD logo,  and presented it to Dr Carruthers at his annual check-up last week.  Doug is thankful for all this specialists and therapists but took the recent opportunity to thank his heart specialist for using heart medications to reverse his cardiomyopathy for over 15 years.  
Deb Robins's insight:
Thank you to all our experts at the front lines of Care on World Duchenne Awareness Day.
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The Heart is a Muscle Too #WDAD18

The Heart is a Muscle Too #WDAD18 | Duchenne Nation Research News | Scoop.it
Cardiomyopathy affects almost every one. 
Deb Robins's insight:
Duchenne hearts can be strengthened. Be proactive and use Standards of Care.
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Keep in Shape with Physiotherapy  #WDAD18

Keep in Shape with Physiotherapy  #WDAD18 | Duchenne Nation Research News | Scoop.it
Any preservation from long term steroid use won't replace daily stretching. 
Deb Robins's insight:
Learn all stretches here: https://youtu.be/6eHLt3KAOtg
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What is the incidence of Duchenne?

What is the incidence of Duchenne? | Duchenne Nation Research News | Scoop.it
Like life expectancy, why not update our background information to the latest statistical information, using the data gathered by national registries in all countries, and the most credible research published?  Patients access journal articles and should be aware of new paradigms flowing from recent information. 
Deb Robins's insight:
Only today saw science articles citing 300,000 worldwide and 1  in every 7,250 males aged 5 to 24
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Thankful for Respiratory Standards  #WDAD18

Thankful for Respiratory Standards  #WDAD18 | Duchenne Nation Research News | Scoop.it
We advocated against outdated thinking, shared journal articles, brought in overseas experts to inspire our specialists and invested in equipment for data to improve our respiratory standards of care. Publishing the current practices makes advocacy easier. #WDAD18
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#WDAD18 Manifesto in French

#WDAD18 Manifesto in French | Duchenne Nation Research News | Scoop.it
#WDAD18  Duchenne awareness post begins at the beginning with the aspirations of the International community for all patients with Duchenne muscular dystrophy.
Deb Robins's insight:
September 1st marks only one week until #WDAD18 (Sept 7th) and Duchenne Nation is posting a resource or opinion every day this month. This is a truly International Day and today's image translated into French language is in honour of Michel Toussaint, the world's #1 DMD respiratory expert.
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CRISPR halts Duchenne muscular dystrophy progression in dogs | EurekAlert! Science News

CRISPR halts Duchenne muscular dystrophy progression in dogs | EurekAlert! Science News | Duchenne Nation Research News | Scoop.it
Scientists for the first time have used CRISPR gene editing to halt the progression of Duchenne muscular dystrophy (DMD) in a large mammal, according to a study by UT Southwestern that provides a strong indication that a lifesaving treatment may be in the pipeline.
Deb Robins's insight:
Promising Dog research that is a very long way off for Humans but the media aren't interested in the lack of data or the inevitable hurdles - naturally.
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Quest for Duchenne Treatment Leads to Breast Cancer Medicine Tamoxifen

Quest for Duchenne Treatment Leads to Breast Cancer Medicine Tamoxifen | Duchenne Nation Research News | Scoop.it
Gavin Ward, a spunky 8-year-old Arkansas boy with Duchenne, enjoys building contraptions with his Lego set and watching video games. He also walks up to 18,000 steps a day and accompanies his fathe…
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The Heart of the Matter - PPMD's Research Priorities 

PPMD is raising $550,000 to continue funding a ongoing Cardiac Initiative. Micro-dystrophin isn't looking enough for hearts, so will need to be augmented by other means or develop a specific micro-dystrophin to correct calcium dysfunction to rescue cardiomyopathy.

Deb Robins's insight:
A panel of experts comment on carrier heart research, the importance of cardiac MRI and measuring circumferential strain & the impact of gene therapy in animal model.
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Sarepta Touts New Data And A “Signal” With Duchenne Gene Therapy

Sarepta Touts New Data And A “Signal” With Duchenne Gene Therapy | Duchenne Nation Research News | Scoop.it

For the first time, a gene therapy---a one-time, long-lasting treatment---has shown it may improve the lives of kids with DMD.

Deb Robins's insight:
Seemingly fantastic levels of micro-dystrophin  but this comment a realistic functional  caveat. "What’s more, a 2015 study on NSAA scores published in the Journal of Neurology, Neurosurgery & Psychiatry noted the motor function of boys under 7 can improve, particularly if treated with steroids at a young age. The fact that the patients in Sarepta’s trial were on steroids could “cloud the picture” a bit, says UPenn’s Elman."
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What is Research if not Curiosity?

What is Research if not Curiosity? | Duchenne Nation Research News | Scoop.it
95% of animal tests shown to be safe and effective, fail in humans. [NIH]  
Deb Robins's insight:
It's about time we did things differently aiming at success via confirmation rather than hoping against failure.
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Go Overboard (in the water) to Exercise

Go Overboard (in the water) to Exercise | Duchenne Nation Research News | Scoop.it
"Use it or lose it" doesn't apply to Duchenne. Think smarter to conserve energy and muscle while expanding the lungs.
Deb Robins's insight:
Know limitations and plan exercise.
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Duchenne Care Videos or Innovative Explanimations? #WDAD18

Duchenne Care Videos or Innovative Explanimations? #WDAD18 | Duchenne Nation Research News | Scoop.it
With good care, life’s prospects, expectations and quality can improve greatly for Duchenne patients worldwide. It is important that patients themselves as well as their families are aware of all they can do to improve their health and their future, for most Duchenne care takes place at home. We produced this series of videos to make this information available to as many people as possible.
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DMD Heart Awareness #WDAD18

DMD Heart Awareness #WDAD18 | Duchenne Nation Research News | Scoop.it
Be proactive about looking after your heart.
Deb Robins's insight:
Don't accept decline in heart function, there are many tools in the Cardiologist's armoury. 
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Orthoses and DMD #WDAD

Orthoses and DMD #WDAD | Duchenne Nation Research News | Scoop.it
AFOs | Piktochart Visual Editor
Deb Robins's insight:
Orthoses help with maximum range during the night.
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Managing your own care  #WDAD18

Managing your own care  #WDAD18 | Duchenne Nation Research News | Scoop.it

Mental outlook is believed to play a role in life expectancy. Take charge of your standards of care.

Deb Robins's insight:
Standards of care can vary even within a single country. Be an advocate.
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Evolutionary Duchenne Care has done the hard yards

Evolutionary Duchenne Care has done the hard yards | Duchenne Nation Research News | Scoop.it
Few authors deviate from outdated incidences and life expectancy in order to frame their own labours. The gains made and shared by the best clinicians and therapists who have extended lives with more quality for so many adults, should be acknowledged verbally. #WDAD18
Deb Robins's insight:
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DMD & Swallowing Food   #WDAD18

DMD & Swallowing Food   #WDAD18 | Duchenne Nation Research News | Scoop.it
Improved standards of care lengthen lifespan, requiring a wider knowledge about common adult issues. One of them is swallowing and these insights by M. Toussaint explain one possible reason behind feeling full,  reduced food intake etc.  A thorough knowledge generates more effective solutions. Talking about progressive weakness isn't as helpful as strategies which can assist with the problem of food and saliva residues in the pyiform sinus pockets, which is a common culprit.
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Pfizer abandons antibody for Duchenne - PMLiVE

Pfizer abandons antibody for Duchenne - PMLiVE | Duchenne Nation Research News | Scoop.it

Latest to fail in myostatin inhibitor class

Deb Robins's insight:
No endpoints reached but Pfizer has more irons in fire.
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Mallinckrodt Announces First Patient Enrolled in Phase 2 Study of MNK-1411 in Duchenne Muscular Dystrophy

Mallinckrodt Announces First Patient Enrolled in Phase 2 Study of MNK-1411 in Duchenne Muscular Dystrophy | Duchenne Nation Research News | Scoop.it

PPMD is excited to learn that Mallinckrodt enrolled the first patient in their Phase 2 study assessing the efficacy and safety of the investigational drug MNK-1411 (cosyntropin, also known as tetracosactide). 

Deb Robins's insight:
Another re-purposed compound. If only extending the label was incentive of itself.
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