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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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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2018 PPMD Conference - Friday, June 29th - Morning Session

LIVE POLLING If you are a patient, caregiver, or family member please go to http://PollEv.com/PPMD to participate live. The 2018 PPMD Annual Conferenc
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2018 PPMD Conference - Thursday, June 28th - Afternoon Session

The 2018 PPMD Annual Conference marks the 24th time families, physicians, researchers, caregivers, industry partners, and those living with Duchenne have gathered to discuss all topics.

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Pulmonary Care in Adults with Duchenne Webinar 28 Mar 1pm Registration

Pulmonary Care in Adults with Duchenne Webinar 28 Mar 1pm Registration | Duchenne Nation Research News | Scoop.it
Deb Robins's insight:
Lisa Wolfe is presenting.
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Duchenne Muscular Dystrophy: As Survival Increases, New Focus on Quality of Life

Duchenne Muscular Dystrophy: As Survival Increases, New Focus on Quality of Life | Duchenne Nation Research News | Scoop.it
Deb Robins's insight:
With thousands of men living into their 30’s and 40’s precisely due to an expectation of survival with quality of life, honed by experienced hands in cardiology, respiratory, medical and therapy arenas; I don’t see the need to be humble by pointing for inspiration to hazy genetic and molecular therapies perhaps legitimized by existing care, genotype, study design and arbitrary measures – nor should such be the justification for better whole-of-life support. Take a bow doctors. The reflection in the mirror of every adult with DMD, is all down to your hands, whether in ideal multi-disciplinary settings or in isolation.
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Duchenne Nation Research News

Duchenne Nation Research News | Duchenne Nation Research News | Scoop.it
Newswire articles about Duchenne muscular dystrophy research, treatment and education.
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Combined use of protein biomarkers and network analysis unveils deregulated regulatory circuits in DMD

Combined use of protein biomarkers and network analysis unveils deregulated regulatory circuits in DMD | Duchenne Nation Research News | Scoop.it

Although the genetic basis of Duchenne muscular dystrophy has been known for almost thirty years, the cellular and molecular mechanisms characterizing the disease are not completely understood and an efficacious treatment remains to be developed. In this study we analyzed proteomics data obtained with the SomaLogic technology from blood serum of a cohort of patients and matched healthy subjects. We developed a workflow based on biomarker identification and network-based pathway analysis that allowed us to describe different deregulated pathways. In addition to muscle-related functions, we identified other biological processes such as apoptosis, signaling in the immune system and neurotrophin signaling as significantly modulated in patients compared with controls. Moreover, our network-based analysis identified the involvement of FoxO transcription factors as putative regulators of different pathways. On the whole, this study provided a global view of the molecular processes involved in Duchenne muscular dystrophy that are decipherable from serum proteome. Read more...

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Application of Quantitative Pharmacology Approaches in Bridging Pharmacokinetics and Pharmacodynamics of Domagrozumab From Adult Healthy Subjects to Pediatric Patients With DMD

Application of Quantitative Pharmacology Approaches in Bridging Pharmacokinetics and Pharmacodynamics of Domagrozumab From Adult Healthy Subjects to Pediatric Patients With DMD | Duchenne Nation Research News | Scoop.it
In summary, quantitative approaches were instrumental in bridging and derisking the fast‐track development of domagrozumab in DMD. Read more...
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Earlier Start of Corticosteroids in DMD Linked to Earlier Heart Disease

Earlier Start of Corticosteroids in DMD Linked to Earlier Heart Disease | Duchenne Nation Research News | Scoop.it
Read about the association between the age when treatment with corticosteroids was initiated and clinical outcomes in Duchenne muscular dystrophy patients.
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Interesting.
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Can Genome Editing Improve the Outlook for Muscular Dystrophy? 

Can Genome Editing Improve the Outlook for Muscular Dystrophy?  | Duchenne Nation Research News | Scoop.it

Researchers investigate genome editing using CRISPR/Cas9 as a way to correct mutations related to Duchenne muscular dystrophy. This approach offers hope to the many patients suffering from Duchenne muscular dystrophy. Human clinical trials have received approvals in China and the United States. However, more research is needed to make sure these genome edits do not cause unexpected mutations outside of their target areas.

Deb Robins's insight:
Could be,  but it isn't right around the corner.
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Duchenne and Becker muscular dystrophy in adolescents: current perspectives

Duchenne and Becker muscular dystrophy in adolescents: current perspectives | Duchenne Nation Research News | Scoop.it

 The increasing proportion of individuals with DMD and BMD living into adulthood underscores the need for more research into interventions and intracacies of adolescence that can improve the social aspects of their lives. 

Deb Robins's insight:
Optimism fades (vainglorious in human terms) and our progress is indebted to the practicalities of care and those advancing it.
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MoveDMD®: Positive Effects of Edasalonexent, an NF-κB Inhibitor, in 4 to 7-Year Old Patients with DMD in Phase 2 Study with an Open-Label Extension

MoveDMD®: Positive Effects of Edasalonexent, an NF-κB Inhibitor, in 4 to 7-Year Old Patients with DMD in Phase 2 Study with an Open-Label Extension | Duchenne Nation Research News | Scoop.it
Slides presented at Action Duchenne International Conference | November 10-12, 2017  UK
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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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2018 PPMD Conference - Thursday, June 28th - Morning Session

The 2018 PPMD Annual Conference marks the 24th time families, physicians, researchers, caregivers, industry partners, and those living with Duchenne have assembled to discuss all current topics.

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Gene Therapy | Duchenne | Sarepta Therapeutics

Gene Therapy | Duchenne | Sarepta Therapeutics | Duchenne Nation Research News | Scoop.it
Learn more about micro-dystrophin and GALGT2 gene therapies for Duchenne muscular dystrophy. Sarepta Therapeutics is dedicated to developing effective therapies to treat 100% of patients with Duchenne.
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What is a Master Protocol? - PPMD Webinar March 2018

In this webinar, PPMD provides basic information regarding the Master Protocol concept, its pros and cons, and how it might impact families. Find out more...

Deb Robins's insight:
Interesting proposal for the purpose of expediting new treatments and for keeping the requisite placebo cohort as small as practicable. A complex approach not easily grasped right away. 
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Reversible immortalisation enables genetic correction of human muscle progenitors and engineering of next-generation human artificial chromosomes for DMD

Finally, we combined the aforementioned functions into a next-generation HAC capable of delivering reversible immortalisation, complete genetic correction, additional dystrophin expression, inducible differentiation and controllable cell death. This work establishes a novel platform for complex gene transfer into clinically relevant human muscle progenitors for DMD gene therapy. Read more...
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New CDC Guidelines Address Emerging Needs of Duchenne Patients

New CDC Guidelines Address Emerging Needs of Duchenne Patients | Duchenne Nation Research News | Scoop.it

As new diagnostic tools and treatments help patients with Duchenne muscular dystrophy (DMD) live into adulthood, new challenges arise in areas like education, independence, personal relationships, health, and intimacy. To address the emerging needs of patients, caregivers and physicians of DMD patients, the Centers for Disease Control and Prevention (CDC) revised guidelines to improve patient transitions into their 30s and beyond. They are divided into 3 documents.  Read more...

Deb Robins's insight:
Consensus care is a bare minimum standard and should be updated, as good care is the whole reason we are seeing improved life expectancy. 
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Consensus guidelines on the use of bisphosphonate therapy in children and adolescents

Consensus guidelines on the use of bisphosphonate therapy in children and adolescents | Duchenne Nation Research News | Scoop.it
The vast majority of corticosteroid-treated boys with DMD do not enter puberty spontaneously, and we would recommend consideration of pubertal induction by age 14.  Read more....
Deb Robins's insight:
An Australian consensus document.
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Expression profiling of disease progression in canine model of DMD

Expression profiling of disease progression in canine model of DMD | Duchenne Nation Research News | Scoop.it

Novel findings include the strong up-regulation of chitinase 3-like 1 (CHI3L1) in faster-progressing GRMD dogs, suggesting previously unexplored mechanisms underlie progression speed in GRMD and DMD. In summary, our findings support the utility of RNA sequencing for evaluating potential biomarkers of GRMD progression speed, and are valuable for identifying new avenues of exploration in DMD research. Read more...

Deb Robins's insight:
More knowledge to shine light on differences in progression that could possibly identify novel therapies.
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Ambulatory capacity in Japanese patients with DMD

Ambulatory capacity in Japanese patients with DMD | Duchenne Nation Research News | Scoop.it
Among 418 patients diagnosed with DMD, 145 patients underwent the 10-meter run/walk test between March 1999 and July 2015. The median age at loss of 10-meter walking ability was 10.4 (interquartile range: 9.2–11.3) years. The 10-meter run/walk speed began to decline 3 years before the loss of 10-meter walking ability, and the median was <1 m/s 1 year before the loss of 10-meter walking ability. Read more...
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Catabasis Pharmaceuticals Reports Additional Data

Catabasis Pharmaceuticals Reports Additional Data | Duchenne Nation Research News | Scoop.it
Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), reports financial results for the fourth quarter and full year ended December 31, 2017, and reviews bus
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Nanostructures created by UCLA scientists could make gene therapies safer, faster and more affordable 

Nanostructures created by UCLA scientists could make gene therapies safer, faster and more affordable  | Duchenne Nation Research News | Scoop.it

UCLA scientists have developed a new method that utilizes microscopic splinter-like structures called “nanospears” for the targeted delivery of biomolecules such as genes straight to patient cells. These magnetically guided nanostructures could enable gene therapies that are safer, faster and more cost-effective.The new method uses 'nanospears' to deliver genes directly to patient cells. Gene therapy has shown great promise as a treatment for a host of diseases.

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FDA slaps a hold on Solid Bio’s gene therapy for Duchenne MD in wake of safety alert

FDA slaps a hold on Solid Bio’s gene therapy for Duchenne MD in wake of safety alert | Duchenne Nation Research News | Scoop.it

Just a few weeks after Solid Biosciences $SLDB put out an 11th hour notice that its gene therapy for Duchenne muscular dystrophy had been put on a partial clinical hold due to safety concerns, just ahead of its $125 million IPO, the biotech is back Wednesday evening with the news that the agency has now halted the study in the wake of an adverse reaction to the therapy by the first patient in the trial.

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