Results 181 to 190 of about 206,878 (242)

Navigating the Prescription Drug Information System

Clinical Pharmacology &Therapeutics, Volume 120, Issue 4, Page 855-870, October 2026.
Information on new prescription drugs is increasingly complex and fragmented, posing challenges for healthcare professionals, patients, and payers. Clinicians require concise, actionable guidance to support prescribing decisions, while patients seek to balance benefits and harms when making decisions aligned with their treatment goals.
Irina V. Wang, Courtney Davis, Robin Forrest, Avi Cherla, Maximilian Siebert, Janice Cowden, Sophia Walker, Tamar Krishnamurti, Florence T. Bourgeois, Steven Woloshin, Anita K. Wagner, Huseyin Naci   +11 more
wiley   +1 more source

MicroRNA–mRNA Networks in Skeletal Muscle of Tailored Pig Models for Dystrophinopathies

Journal of Cachexia, Sarcopenia and Muscle, Volume 17, Issue 5, October 2026.
ABSTRACT Background Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) are X‐linked dystrophinopathies caused by mutations in the dystrophin (DMD) gene. A common DMD‐causing mutation in humans is exon 52 deletion (DMDΔ52), which disrupts the reading frame and abolishes dystrophin expression.
Sarah Reschke, Alexander Graf, Frieder Hadlich, Florian Jaudas, Thomas Fröhlich, Michael Stirm, Nikolai Klymiuk, Stefan Krebs, Eckhard Wolf, Asghar Ali   +9 more
wiley   +1 more source

Genetic and Pharmacologic Inhibition of Myostatin Restores Muscle Mass in a Dynamin 2‐Related Centronuclear Myopathy Mouse Model

Journal of Cachexia, Sarcopenia and Muscle, Volume 17, Issue 5, October 2026.
ABSTRACT Background Autosomal dominant centronuclear myopathy (ADCNM), most commonly caused by mutations in the dynamin 2 (DNM2) gene, is a rare congenital myopathy characterized by progressive muscle weakness and atrophy. Myostatin, a key negative regulator of skeletal muscle mass, has shown therapeutic potential in several models of neuromuscular ...
Durieux Anne‐Cécile, Arnould David, Velarde Mathias, Allibert Valentine, Paret Chloé, Debelley Quentin, Kizilkilic Esra, Chambon Sidney, Girard Emmanuelle, Collin Louise, Gensel Alexandre, Castells Josiane, Pasternack Aja, Bowen Maximilien, Morel Baptiste, Hourdé Christophe, Laurent Schaeffer, Ritvos Olli, Bitoun Marc, Mounier Rémi, Freyssenet Damien   +20 more
wiley   +1 more source

Quantitative Muscle MRI Fat Fraction as a Biomarker of Disease Severity in Mitochondrial Myopathies

Journal of Cachexia, Sarcopenia and Muscle, Volume 17, Issue 5, October 2026.
ABSTRACT Background Quantitative muscle MRI is increasingly used to assess structural muscle damage in inherited myopathies, but its application in primary mitochondrial myopathies (PMM) has not been systematically evaluated in large cohorts. Because PMM are clinically and genetically heterogeneous, objective imaging biomarkers are needed to quantify ...
Ana Bermejo‐Moriñigo, Paloma Martín‐Jiménez, Violeta González‐Méndez, Laura Bermejo‐Guerrero, Luz Edith Ochoa, Cristina Martín‐Arriscado, Andrea Alcalá‐Galiano, Cristina Casado‐Pérez, María Navarro‐Riquelme, Rocío Garrido‐Moraga, Adrián González Quintana, Alberto Blázquez, Cristina Domínguez‐González   +12 more
wiley   +1 more source

Concurrent Treatment With Lorlatinib and Dinutuximab Beta Induced Acute Gastric Dilation in a Pediatric Patient With Neuroblastoma

Pediatric Blood &Cancer, Volume 73, Issue 10, October 2026.
Suna Lin, Linjie Li, JiJun Chen, Zhaokun Tao, Lifeng Zhang, Junqing Mao, Jinhu Wang   +6 more
wiley   +1 more source

Teaching Cell and Gene Therapy in Genetic Counseling Programs: Survey Findings and Suggested Practical Resources for Integration

Journal of Genetic Counseling, Volume 35, Issue 5, October 2026.
ABSTRACT As cell and gene therapies (CGTs) advance through clinical trials into clinical practice, it becomes increasingly important for genetic counselors (GCs) to understand these therapeutics to discuss them with patients. Despite the apparent need for GCs to obtain CGT‐related education, little is known about the training GCs receive on CGT topics ...
Mariel Sebby, Lily McConachie, Leah Wetherill, Paula Delk, Emily L. Hopewell   +4 more
wiley   +1 more source

Imaging of Facial Muscles in Facioscapulohumeral Muscular Dystrophy: An Exploratory Study Comparing Magnetic Resonance Imaging and Ultrasound

Muscle &Nerve, Volume 74, Issue 4, Page 1256-1262, October 2026.
ABSTRACT Aims Facioscapulohumeral muscular dystrophy (FSHD) is a genetic progressive muscle disorder often presenting with facial weakness. However, imaging studies specifically evaluating facial muscle involvement and its relationship with clinical severity remain limited. This preliminary study explored magnetic resonance imaging (MRI) and ultrasound
Federico Pistoia, Elena Faedo, Marta Macciò, Giovanni Marcenaro, Maria Elena Susi, Mehrnaz Hamedani, Veronica Caramellino, Sara Massucco, Andrea Francesco Maria Vollaro, Paola Mandich, Fabio Gotta, Riccardo Picasso, Federico Zaottini, Marina Grandis, Carlo Martinoli   +14 more
wiley   +1 more source

A Multidisciplinary Approach to the Anesthetic Management of Patients With Duchenne Muscular Dystrophy

Pediatric Pulmonology, Volume 61, Issue 10, October 2026.
ABSTRACT Duchenne muscular dystrophy (DMD) is an inherited neuromuscular disorder with multisystem involvement that causes progressive muscular weakness and cardiorespiratory dysfunction. The first comprehensive recommendations regarding anesthetic management are now almost 20 years old and, in the interval, DMD care has evolved.
Fabrizio Racca, Elena Conti, Giulia Racca, Luigi Barberis, David J. Birnkrant   +4 more
wiley   +1 more source

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Muscular Dystrophies

Clinics in Chest Medicine, 2018
Muscular dystrophies represent a complex, varied, and important subset of neuromuscular disorders likely to require the care of a pulmonologist. The spectrum of conditions encapsulated by this subset ranges from severe and fatal congenital muscular dystrophies with onset in infancy to mild forms of limb and girdle weakness with onset in adulthood and ...
John C, Carter, Daniel W, Sheehan, Andre, Prochoroff, David J, Birnkrant   +3 more
openaire   +2 more sources

Muscular dystrophies

Current Opinion in Neurology, 2002
Muscular dystrophy includes many genetically distinct disorders. The list of causative genes for muscular dystrophy has been expanding rapidly, including those for congenital muscular dystrophies.We review the newly identified causative genes and suggested molecular mechanisms, focusing on glycosylation abnormality of alpha-dystroglycan, collagen VI ...
Ichizo, Nishino, Eijiro, Ozawa
openaire   +2 more sources