Results 61 to 70 of about 73,251 (284)

Duchenne muscular dystrophy: current cell therapies

Therapeutic Advances in Neurological Disorders, 2015
Duchenne muscular dystrophy is a genetically determined X-linked disease and the most common, progressive pediatric muscle disorder. For decades, research has been conducted to find an effective therapy.
Dorota Sienkiewicz, Wojciech Kulak, Bożena Okurowska-Zawada, Grażyna Paszko-Patej, Katarzyna Kawnik   +4 more
doaj   +1 more source

Myofibre Density Reveals a Critical Threshold Around Age 6 in Steroid-Naïve Duchenne Muscular Dystrophy: A Retrospective Observational Study. [PDF]

Neuropathol Appl Neurobiol
We analysed archival muscle biopsies of steroid‐naïve patients with Duchenne muscular dystrophy (DMD), proposing a novel, simple metric: myofibre density (MFD). MFD sharply declines until age 6, identifying a critical threshold around this age. MFD provides a sensitive, reproducible biomarker for early disease progression, highlighting a potential ...
Yamakado T, Ishikawa Y, Ise K, Wang L, Oda Y, Tsuda M, Kimura T, Nagao M, Ito M, Tanaka S, Tanei ZI.   +10 more
europepmc   +2 more sources

Optimization of CEST MRI Reporter Protein Design Using Cation‐Pi Networks

Chemistry – A European Journal, EarlyView.
A novel engineering approach can produce reporter proteins for cell and viral therapy tracking with unique magnetic resonance imaging (MRI) signatures, detectable with chemical exchange saturation transfer (CEST). We discover how cation‐π interactions between amino acid groups can help us fine‐tune magnetic resonance properties for noninvasive ...
David E. Korenchan, Ethan J. French, Emerenziana Runco, Chetan B. Dhakan, Jinwu Yan, Hiroshi Nakashima, Michael T. McMahon, Assaf A. Gilad, Christian T. Farrar   +8 more
wiley   +1 more source

Exosome-Mediated Benefits of Cell Therapy in Mouse and Human Models of Duchenne Muscular Dystrophy

Stem Cell Reports, 2018
Summary: Genetic deficiency of dystrophin leads to disability and premature death in Duchenne muscular dystrophy (DMD), affecting the heart as well as skeletal muscle.
Mark A. Aminzadeh, Russell G. Rogers, Mario Fournier, Rachel E. Tobin, Xuan Guan, Martin K. Childers, Allen M. Andres, David J. Taylor, Ahmed Ibrahim, Xiangming Ding, Angelo Torrente, Joshua M. Goldhaber, Michael Lewis, Roberta A. Gottlieb, Ronald A. Victor, Eduardo Marbán   +15 more
doaj   +1 more source

Prognosis of Right Ventricular Systolic Dysfunction in Patients With Duchenne Muscular Dystrophy

Journal of the American Heart Association: Cardiovascular and Cerebrovascular Disease, 2023
Background Chronic respiratory failure and heart involvement may occur in Duchenne muscular dystrophy. We aimed to assess the prognostic value of the right ventricular (RV) systolic dysfunction in patients with Duchenne muscular dystrophy.
Abdallah Fayssoil, Nicolas Mansencal, Lee S. Nguyen, Olivier Nardi, Rabah Ben Yaou, France Leturcq, Helge Amthor, Karim Wahbi, Henri Marc Becane, Frederic Lofaso, Helene Prigent, Guillaume Bassez, Anthony Behin, Tanya Stojkovic, Bertrand Fontaine, Denis Duboc, Olivier Dubourg, Bernard Clair, Pascal Laforet, Djillali Annane, David Orlikowski   +20 more
doaj   +1 more source

Dasatinib as a treatment for Duchenne muscular dystrophy [PDF]

, 2016
Identification of a systemically acting and universal small molecule therapy for Duchenne muscular dystrophy would be an enormous advance for this condition.
Emmerson, T., Lipscomb, L., Piggott, R.W., Winder, S.J.   +3 more
core   +1 more source

Pediatric Neuropalliative Medicine Clinic: Five‐Year Data Characterizing a Novel Model of Outpatient Care

Annals of the Child Neurology Society, EarlyView.
ABSTRACT Objective Pediatric neuropalliative medicine (PNPM) is a recently developed area of subspecialty neurology practice focused on supporting the complex emotional, psychological, and physical aspects of caring for a child with serious neurological disease.
Lauren Treat, Lori Silveira, Kevin C. Ess   +2 more
wiley   +1 more source

Elevation of transaminases. What if not the liver?

Лечащий Врач
Background. According to Russian studies, the average age of Duchenne muscular dystrophy diagnosis is 7-8 years. This is because, on one hand, Duchenne muscular dystrophy is a rare disease, and a doctor may never see it throughout their clinical practice.
I. V. Sharkova
doaj   +1 more source

Fhod3 in zebrafish supports myofibril stability during growth of embryonic skeletal muscle

Developmental Dynamics, EarlyView.
Abstract Background Actin filament organization in cardiomyocytes critically depends on the formin Fhod3, but a role for Fhod3 in skeletal muscle development has not yet been described. Results We demonstrate here that in zebrafish mutated for one of two fhod3 paralog genes, fhod3a, skeletal muscle of the trunk appears normal through 2 days post ...
Aubrie Russell, Tracy Eng, Jeffrey D. Amack, David Pruyne   +3 more
wiley   +1 more source

Duchenne muscular dystrophy

, 2017
Duchenne muscular dystrophy is a genetic disease inherited in an X-linked recessive pattern and characterized by the lack of the protein dystrophin. Boys with the genetic defect show symptoms of the disease at a young age, and symptoms progressively worsen and result in an early death.
Rohit Sharma, Arlene Campos, Craig Hacking   +2 more
  +4 more sources