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About 5,718 results

ALLMedicine™ Duchenne Muscular Dystrophy Center

Research & Reviews  2,037 results

The association between cardiac involvement and long-term clinical outcomes in patients...
https://doi.org/10.1002/ehf2.13970
ESC Heart Failure; Cha JJ, Kim IS et. al.

May 18th, 2022 - Despite advances in contemporary cardiopulmonary therapies, cardiomyopathy remains the leading cause of death in patients with Duchenne muscular dystrophy (DMD). Also, the long-term clinical outcomes of patients with DMD and cardiomyopathy is unkn...

Casimersen for the treatment of Duchenne muscular dystrophy.
https://doi.org/10.1016/j.tips.2022.04.009
Trends in Pharmacological Sciences; Zakeri SE, Pradeep SP et. al.

May 18th, 2022 - Casimersen for the treatment of Duchenne muscular dystrophy.|2022|Zakeri SE,Pradeep SP,Kasina V,Laddha AP,Manautou JE,|

Obesity and loss of ambulation are associated with lower extremity oedema in Duchenne m...
https://doi.org/10.1017/S1047951122001342
Cardiology in the Young; Freytag JM, Ryan TD et. al.

May 14th, 2022 - Patients with Duchenne muscular dystrophy have multiple risk factors for lower extremity oedema. This study sought to define the frequency and predictors of oedema. Patients aged 15 years and older were screened by patient questionnaire, and the p...

Age at Diagnosis for Duchenne Muscular Dystrophy: Why we must do better.
https://doi.org/10.1002/mus.27574
Muscle & Nerve; Quinlivan RM

May 14th, 2022 - Age at Diagnosis for Duchenne Muscular Dystrophy: Why we must do better.|2022|Quinlivan RM,|

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Guidelines  6 results

2022 HRS expert consensus statement on evaluation and management of arrhythmic risk in ...
https://doi.org/10.1016/j.hrthm.2022.04.022
Heart Rhythm; Groh WJ, Bhakta D et. al.

May 3rd, 2022 - This international multidisciplinary document is intended to guide electrophysiologists, cardiologists, other clinicians, and health care professionals in caring for patients with arrhythmic complications of neuromuscular disorders (NMDs). The doc...

Standardized Data Structures in Rare Diseases: CDISC User Guides for Duchenne Muscular ...
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7877853
Clinical and Translational Science; Mullin AP, Corey D et. al.

Jul 24th, 2020 - Interest in drug development for rare diseases has expanded dramatically since the Orphan Drug Act was passed in 1983, with 40% of new drug approvals in 2019 targeting orphan indications. However, limited quantitative understanding of natural hist...

Respiratory Management of the Patient With Duchenne Muscular Dystrophy.
https://doi.org/10.1542/peds.2018-0333H
Pediatrics Sheehan DW, Birnkrant DJ et. al.

Oct 3rd, 2018 - In 2010, Care Considerations for Duchenne Muscular Dystrophy, sponsored by the Centers for Disease Control and Prevention, was published in Lancet Neurology, and in 2018, these guidelines were updated. Since the publication of the first set of gui...

Consensus guidelines on the use of bisphosphonate therapy in children and adolescents.
https://doi.org/10.1111/jpc.13768
Journal of Paediatrics and Child Health; Simm PJ, Biggin A et. al.

Mar 6th, 2018 - Bisphosphonate therapy is the mainstay of pharmacological intervention in young people with skeletal fragility. The evidence of its use in a variety of conditions remains limited despite over three decades of clinical experience. On behalf of the ...

Contemporary cardiac issues in Duchenne muscular dystrophy. Working Group of the Nation...
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4573596
Circulation McNally EM, Kaltman JR et. al.

May 6th, 2015 - Contemporary cardiac issues in Duchenne muscular dystrophy. Working Group of the National Heart, Lung, and Blood Institute in collaboration with Parent Project Muscular Dystrophy.|2015|McNally EM,Kaltman JR,Benson DW,Canter CE,Cripe LH,|therapeuti...

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Drugs  8 results see all →

Clinicaltrials.gov  87 results

A Study to Evaluate the Safety and Tolerability of PF-06939926 Gene Therapy in Duchenne Muscular Dystrophy
https://clinicaltrials.gov/ct2/show/NCT03362502

Apr 27th, 2022 - This is a first-in-human/first-in-patient, multi-center, open-label, non-randomized, ascending dose, safety and tolerability study of a single intravenous infusion of PF-06939926 in ambulatory and non-ambulatory subjects with Duchenne muscular dys...

Gene Transfer Clinical Trial to Deliver rAAVrh74.MCK.GALGT2 for Duchenne Muscular Dystrophy
https://clinicaltrials.gov/ct2/show/NCT03333590

Apr 25th, 2022 - This is an open-label, dose escalation trial where the vector will be delivered via the femoral artery to the muscles of both legs of DMD subjects. The primary objective of this study is the assessment of the safety of intravascular administration...

Bicycle Ergometer Training in Duchenne Muscular Dystrophy
https://clinicaltrials.gov/ct2/show/NCT05330195

Apr 15th, 2022 - The progressive muscle weakness and contractures of the patients adversely affect their gait and balance. It is known that the disorder of the patients' balance and gait affects their functional capacity. The aim of this study is to examine the ef...

A Study to Compare Safety and Efficacy of a High Dose of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) (MIS51ON)
https://clinicaltrials.gov/ct2/show/NCT03992430

Apr 12th, 2022 - This study will be comprised of 2 parts: Part 1 (dose escalation) will be conducted to evaluate the safety and tolerability of 2 doses (high dose level 1 and high dose level 2) of eteplirsen in approximately 10 participants with DMD; Part 2 (dose ...

Exploratory Study of NS-089/NCNP-02 in DMD
https://clinicaltrials.gov/ct2/show/NCT04129294

Apr 11th, 2022 - This study is designed to assess the safety, tolerability, efficacy and pharmacokinetics (PK) of NS-089/NCNP-02 in subjects diagnosed with Duchenne muscular dystrophy (DMD), and to determine the dosage for subsequent studies.

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News  144 results

Five New Neurology Studies: Possible Breakthroughs, Some Failures
https://www.medscape.com/viewarticle/972485

May 9th, 2022 - This transcript has been edited for clarity. Dear colleagues, I'm Christoph Diener from the University of Duisburg-Essen in Germany. Today, I would like to report on five exciting studies from March 2022. Cell Therapy for Duchenne Muscular Dystrop...

Tau Moderated by Menopause; CTE Risk Underestimated; When Is a Single Study Enough?
https://www.medpagetoday.com/neurology/generalneurology/98523

May 3rd, 2022 - Menopause status moderated sex differences in tau burden, with a divergence in tauopathy between the sexes apparent 20 years earlier than previously reported. (Annals of Neurology) Plasma p-tau was a less consistent marker of brain amyloidosis in ...

First Human Bird Flu Case in U.S.; Okla. Nears Abortion Ban; Ebola Outbreak in DRC
https://www.medpagetoday.com/infectiousdisease/covid19/98463

Apr 29th, 2022 - Note that some links may require registration or subscription. CDC confirmed the first human case of the H5N1 bird flu in the U.S., in a Colorado man with direct exposure to birds. Oklahoma Gov. Kevin Stitt (R) is expected to sign a "Texas-style" ...

Optimal Treatment Regimen for Muscular Dystrophy Identified?
https://www.medscape.com/viewarticle/971703

Apr 6th, 2022 - Daily corticosteroid use is significantly more effective than an intermittent regimen for improving motor function in patients with Duchenne muscular dystrophy (DMD), a new study suggests. Dr Michela Guglieri "The intermittent regimen has been use...

A Useful Severity Predictor in Muscular Dystrophy?
https://www.medscape.com/viewarticle/969943

Mar 9th, 2022 - The study covered in this summary was published on Researchsquare.com as a preprint and has not yet been peer reviewed. Key Takeaways Peripheral eosinophil count was negatively correlated with the Vignos muscle strength score in pediatric patients...

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Patient Education  5 results see all →