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About 5,908 results

ALLMedicine™ Duchenne Muscular Dystrophy Center

Research & Reviews  2,094 results

Using machine learning algorithms to enhance the diagnostic performance of electrical i...
https://doi.org/10.1002/mus.27664
Muscle & Nerve; Pandeya SR, Nagy JA et. al.

Jun 22nd, 2022 - We assessed the classification performance of machine learning (ML) using multifrequency electrical impedance myography (EIM) values to improve upon diagnostic outcomes as compared to those based on a single EIM value. EIM data was obtained from u...

Delays in pulmonary decline in eteplirsen-treated patients with Duchenne muscular dystr...
https://doi.org/10.1002/mus.27662
Muscle & Nerve; Iff J, Gerrits C et. al.

Jun 19th, 2022 - Pulmonary decline is an important issue in patients with Duchenne muscular dystrophy (DMD). Eteplirsen is a US-approved treatment for patients with DMD and exon 51 skip-amenable mutations. Previous analyses have shown that eteplirsen is associated...

Angiotensin receptor-neprilysin inhibitor in symptomatic patients with Duchenne dilated...
https://doi.org/10.1002/ehf2.13963
ESC Heart Failure; Arcudi A, Di Francesco M et. al.

Jun 18th, 2022 - Duchenne muscular dystrophy (DMD) is an X-linked recessive neuromuscular disorder, characterized by significant long-term cardiac involvement. Dilated cardiomyopathy (DCM) is the main cause of death in DMD, and angiotensin-converting enzyme inhibi...

A Phase 3 Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy
https://clinicaltrials.gov/ct2/show/NCT04281485

Jun 16th, 2022 - The study will assess the efficacy of PF-06939926 gene therapy on ambulatory function while also monitoring its safety. Approximately 99 boys with DMD will be enrolled and randomly assigned to one of two groups: approximately two thirds will be in...

Antioxidants for Treatment of Duchenne Muscular Dystrophy: A Systematic Review and Meta...
https://doi.org/10.1159/000525045
European Neurology; Ren S, Yao C et. al.

Jun 14th, 2022 - Increasing evidence has shown that oxidative stress is involved in the pathogenesis of Duchenne muscular dystrophy (DMD). Oxidative stress impairs muscle function, reduces regenerative capacity, and leads to atrophy and muscle weakness. The presen...

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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  93 results

A Phase 3 Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy
https://clinicaltrials.gov/ct2/show/NCT04281485

Jun 16th, 2022 - The study will assess the efficacy of PF-06939926 gene therapy on ambulatory function while also monitoring its safety. Approximately 99 boys with DMD will be enrolled and randomly assigned to one of two groups: approximately two thirds will be in...

Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Duchenne Muscular Dystrophy
https://clinicaltrials.gov/ct2/show/NCT02851797

Jun 10th, 2022 - Givinostat or placebo oral suspension (10 mg/mL) will be administered orally as 2 oral doses daily while the subject is in fed state, according to the child's weight. Study drug should be permanently stopped if any of the following occur: severe d...

A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From SRP-9001 (Delandistrogene Moxeparvovec) in Participants With Duchenne Muscular Dystrophy (DMD)
https://clinicaltrials.gov/ct2/show/NCT04626674

Jun 8th, 2022 - This is an open-label gene transfer therapy study evaluating the safety of and expression from SRP-9001 (delandistrogene moxeparvovec) in participants with DMD over 260 weeks.

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

May 25th, 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 (100 milligrams/kilogram [mg/kg] and 200 mg/kg) of eteplirsen in approximately 10 participants with DMD; Part 2 ...

A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy (HOPE-3)
https://clinicaltrials.gov/ct2/show/NCT05126758

May 20th, 2022 - Up to 68 eligible study participants will be randomized to either CAP-1002 or placebo in a 1:1 ratio. The trial will include visits at Screening, Baseline/Day 1, Week 4, and Months 3, 6, 9, and 12 with IV infusions of CAP-1002 or placebo on Day 1 ...

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News  157 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 →