ALLMedicine™ Duchenne Muscular Dystrophy Center
Research & Reviews 2,094 results
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...
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...
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...
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...
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...
Guidelines 6 results
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...
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...
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...
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 ...
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...
Drugs 8 results see all →
Clinicaltrials.gov 93 results
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...
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...
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.
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 ...
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 ...
News 157 results
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...
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 ...
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" ...
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...
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...