| Publication |
Sentence |
Publish Date |
Extraction Date |
Species |
| Matthew J Burke, Braiden M Blatt, James A Teixeira, Dennis O Pérez-López, Yongping Yue, Xiufang Pan, Chady H Hakim, Gang Yao, Roland W Herzog, Dongsheng Dua. Adeno-Associated Virus 8 and 9 Myofibre Type/Size Tropism Profiling Reveals Therapeutic Effect of Microdystrophin in Canines. Journal of cachexia, sarcopenia and muscle. vol 16. issue 1. 2025-01-10. PMID:39790021. |
adeno-associated virus (aav) 8 and 9 are in clinical trials for treating neuromuscular diseases such as duchenne muscular dystrophy (dmd). |
2025-01-10 |
2025-01-13 |
Not clear |
| Liping Ge, Yang Yang, Yanfei Yang, Yanfei Chen, Na Tao, Liping Zhang, Canmiao Zhao, Xing Zhan. DMD mutations in pediatric patients with phenotypes of Duchenne/Becker muscular dystrophy. Open medicine (Warsaw, Poland). vol 19. issue 1. 2024-11-26. PMID:39588385. |
duchenne muscular dystrophy (dmd) and becker muscular dystrophy (bmd) are common x-inherited neuromuscular diseases. |
2024-11-26 |
2024-11-28 |
human |
| Craig M McDonald, Jacob S Elkins, Sai Dharmarajan, Katherine Gooch, Teofil Ciobanu, Claire J Lansdall, Alexander P Murphy, Fiona McDougall, Eugenio M Mercuri, Ivana Audhy. Caregiver Global Impression Observations from EMBARK: A Phase 3 Study Evaluating Delandistrogene Moxeparvovec in Ambulatory Patients with Duchenne Muscular Dystrophy. Neurology and therapy. 2024-11-26. PMID:39589719. |
duchenne muscular dystrophy (dmd) is a rare, progressive, debilitating neuromuscular disease. |
2024-11-26 |
2024-11-28 |
Not clear |
| Andrew McClennan, Lisa Hoffma. Angiopoietin 1 Attenuates Dysregulated Angiogenesis in the Gastrocnemius of DMD Mice. International journal of molecular sciences. vol 25. issue 21. 2024-11-09. PMID:39519374. |
duchenne muscular dystrophy (dmd) is a degenerative neuromuscular disease caused by a lack of functional dystrophin. |
2024-11-09 |
2024-11-17 |
mouse |
| Domenico Gorgoglione, Daniele Sabbatini, Pietro Riguzzi, Giuliana Capece, Marika Pane, Serenella Servidei, Marta Briganti, Cristina Sancricca, Fabio Bruschi, Anna Ardissone, Riccardo Masson, Annamaria Gallone, Lorenzo Maggi, Esther Picillo, Luisa Politano, Angela Petrosino, Sara Vianello, Martina Penzo, Matteo Villa, Maria Sframeli, Cosimo Allegra, Andrea Barp, Alessandra Di Bari, Francesca Salmin, Emilio Albamonte, Giovanni Colacicco, Chiara Panicucci, Monica Traverso, Concetta Palermo, Alberto Lerario, Daniele Velardo, Maria G D'Angelo, Angela Berardinelli, Alice Gardani, Roberta Nicotra, Stefano Parravicini, Gabriele Siciliano, Giulia Ricci, Francesca Torri, Giulio Gadaleta, Guido Urbano, Enrica Rolle, Federica Ricci, Adele D'Amico, Michela Catteruccia, Antonella Pini, Melania Giannotta, Roberta Battini, Gemma Marinella, Stefano C Previtali, Alberto A Zambon, Alessandra Ferlini, Fernanda Fortunato, Francesca Magri, Tiziana E Mongini, Valeria A Sansone, Claudio Bruno, Sonia Messina, Vincenzo Nigro, Isabella Moroni, Eugenio Mercuri, Luca Bello, Elena Pegorar. Natural history of Becker muscular dystrophy: DMD gene mutations predict clinical severity. Brain : a journal of neurology. 2024-11-05. PMID:39499670. |
becker muscular dystrophy (bmd) is an x-linked neuromuscular disease due to mutations in the dmd gene, leading to a deficient and less functional dystrophin mainly in skeletal and cardiac muscle. |
2024-11-05 |
2024-11-08 |
Not clear |
| Jerry R Mendell, Francesco Muntoni, Craig M McDonald, Eugenio M Mercuri, Emma Ciafaloni, Hirofumi Komaki, Carmen Leon-Astudillo, Andrés Nascimento, Crystal Proud, Ulrike Schara-Schmidt, Aravindhan Veerapandiyan, Craig M Zaidman, Maitea Guridi, Alexander P Murphy, Carol Reid, Christoph Wandel, Damon R Asher, Eddie Darton, Stefanie Mason, Rachael A Potter, Teji Singh, Wenfei Zhang, Paulo Fontoura, Jacob S Elkins, Louise R Rodino-Klapa. AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trial. Nature medicine. 2024-10-09. PMID:39385046. |
duchenne muscular dystrophy (dmd) is a rare, x-linked neuromuscular disease caused by pathogenic variants in the dmd gene that result in the absence of functional dystrophin, beginning at birth and leading to progressive impaired motor function, loss of ambulation and life-threatening cardiorespiratory complications. |
2024-10-09 |
2024-10-12 |
Not clear |
| Yuki Takanarita, Yuichi Okata, Hiroyuki Awano, Ryosuke Bo, Yuko Bito. Long-term course of gastrostomy nutritional management in patients with Duchenne muscular dystrophy: A retrospective cohort study. Journal of clinical neuroscience : official journal of the Neurosurgical Society of Australasia. vol 129. 2024-10-03. PMID:39362116. |
duchenne muscular dystrophy (dmd) is a progressive neuromuscular disease that commonly requires gastrostomy due to dysphagia. |
2024-10-03 |
2024-10-07 |
Not clear |
| Clémence Kiho Bourgeois Yoshioka, Nana Takenaka-Ninagawa, Megumi Goto, Mayuho Miki, Daiki Watanabe, Masamichi Yamamoto, Tomoki Aoyama, Hidetoshi Sakura. Cell transplantation-mediated dystrophin supplementation efficacy in Duchenne muscular dystrophy mouse motor function improvement demonstrated by enhanced skeletal muscle fatigue tolerance. Stem cell research & therapy. vol 15. issue 1. 2024-09-20. PMID:39300595. |
duchenne muscular dystrophy (dmd) is an incurable neuromuscular disease leading to progressive skeletal muscle weakness and fatigue. |
2024-09-20 |
2024-09-22 |
mouse |
| Ryan Fischer, Pat Furlong, Annie Kennedy, Kelly Maynard, Marissa Penrod, Debra Miller, Chamindra G Laverty, Linda P Lowes, Nancy L Kuntz, Perry B Shieh, Jane Kondejewski, Peter J Neumann, Jason Shafrin, Richard J Willk. Healthcare Stakeholder Perspectives on a Value Assessment Approach for Duchenne Muscular Dystrophy Therapies. Journal of multidisciplinary healthcare. vol 17. 2024-09-03. PMID:39224484. |
traditional value assessment frameworks are challenged in comprehensively assessing the societal value new therapies bring to individuals with rare, progressive, genetic, fatal, neuromuscular diseases such as duchenne muscular dystrophy (dmd). |
2024-09-03 |
2024-09-05 |
Not clear |
| Alexa C Klimchak, Lauren E Sedita, Eleanor M Perfetto, Katherine L Gooch, Daniel C Malon. Discriminatory properties of QALY-based CEA for patients with disabilities: A Duchenne muscular dystrophy case study. Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research. 2024-08-02. PMID:39094692. |
this study examined the impact of a qaly-based assessment on the price for a hypothetical treatment for duchenne muscular dystrophy (dmd), a progressive, genetic neuromuscular disease. |
2024-08-02 |
2024-08-06 |
Not clear |
| Hitham Aldhare. Duchenne muscular dystrophy in Saudi Arabia: a review of the current literature. Frontiers in neurology. vol 15. 2024-08-01. PMID:39087004. |
dmd is a rare, severe neuromuscular disease that causes death due to cardiovascular and respiratory complications among affected boys. |
2024-08-01 |
2024-08-03 |
Not clear |
| Shivam Gandhi, H Lee Sweeney, Cora C Hart, Renzhi Han, Christopher G R Perr. Cardiomyopathy in Duchenne Muscular Dystrophy and the Potential for Mitochondrial Therapeutics to Improve Treatment Response. Cells. vol 13. issue 14. 2024-07-26. PMID:39056750. |
duchenne muscular dystrophy (dmd) is a progressive neuromuscular disease caused by mutations to the dystrophin gene, resulting in deficiency of dystrophin protein, loss of myofiber integrity in skeletal and cardiac muscle, and eventual cell death and replacement with fibrotic tissue. |
2024-07-26 |
2024-07-28 |
Not clear |
| Kalina Andrysiak, Paweł E Ferdek, Anna M Sanetra, Gabriela Machaj, Luisa Schmidt, Izabela Kraszewska, Katarzyna Sarad, Katarzyna Palus-Chramiec, Olga Lis, Marta Targosz-Korecka, Marcus Krüger, Marian H Lewandowski, Guillem Ylla, Jacek Stępniewski, Józef Dula. Upregulation of utrophin improves the phenotype of Duchenne muscular dystrophy hiPSC-derived CMs. Molecular therapy. Nucleic acids. vol 35. issue 3. 2024-07-22. PMID:39035791. |
duchenne muscular dystrophy (dmd) is a genetic neuromuscular disease. |
2024-07-22 |
2024-07-24 |
human |
| Rebecca Delaney, Ken D O'Hallora. Respiratory performance in Duchenne muscular dystrophy: Clinical manifestations and lessons from animal models. Experimental physiology. 2024-07-18. PMID:39023735. |
duchenne muscular dystrophy (dmd) is a fatal genetic neuromuscular disease. |
2024-07-18 |
2024-07-20 |
mouse |
| Camille Verebi, Victor Gravrand, Thierry Bienvenu, France Leturcq, Juliette Nectou. A retrospective cohort study and review of the literature about germline mosaicism in Duchenne/Becker muscular dystrophy prenatal counseling: How to estimate the recurrence risk in clinical settings? Journal of genetic counseling. 2024-06-19. PMID:38895972. |
duchenne muscular dystrophy (dmd) and becker muscular dystrophy (bmd) are the most common inherited neuromuscular diseases. |
2024-06-19 |
2024-06-21 |
Not clear |
| b' Chantal A Coles, Keryn G Woodman, Elizabeth M Gibbs, Rachelle H Crosbie, Jason D White, Shireen R Lamand\\xc3\\xa. Benfotiamine improves dystrophic pathology and exercise capacity in mdx mice by reducing inflammation and fibrosis. Human molecular genetics. 2024-05-06. PMID:38710523.' |
duchenne muscular dystrophy (dmd) is a progressive and fatal neuromuscular disease. |
2024-05-06 |
2024-05-27 |
mouse |
| Christina Qian, Alexa C Klimchak, Shelagh M Szabo, Evan Popoff, Susan T Iannaccone, Katherine L Gooc. Observing the Clinical Course of Duchenne Muscular Dystrophy in Medicaid Real-World Healthcare Data. Advances in therapy. 2024-05-02. PMID:38698169. |
duchenne muscular dystrophy (dmd) is a rare, severe progressive neuromuscular disease. |
2024-05-02 |
2024-05-05 |
Not clear |
| Kay E Davies, Julie Vog. Long-term clinical follow-up of a family with Becker muscular dystrophy associated with a large deletion in the DMD gene. Neuromuscular disorders : NMD. vol 39. 2024-04-23. PMID:38653179. |
duchenne muscular dystrophy is a neuromuscular disease caused by dmd gene mutations that result in an absence of functional dystrophin protein. |
2024-04-23 |
2024-04-26 |
Not clear |
| Craig M Zaidman, Natalie L Goedeker, Amal A Aqul, Russell J Butterfield, Anne M Connolly, Ronald G Crystal, Kara E Godwin, Kan N Hor, Katherine D Mathews, Crystal M Proud, Elizabeth Kula Smyth, Aravindhan Veerapandiyan, Paul B Watkins, Jerry R Mendel. Management of Select Adverse Events Following Delandistrogene Moxeparvovec Gene Therapy for Patients With Duchenne Muscular Dystrophy. Journal of neuromuscular diseases. 2024-04-12. PMID:38607761. |
duchenne muscular dystrophy (dmd) is a rare, degenerative, recessive x-linked neuromuscular disease. |
2024-04-12 |
2024-04-15 |
Not clear |
| Rose Mary Ferreira Lisboa da Silva, Nathalia Mussi Monteze, Juliana Gurgel Giannetti, Zilda Maria Alves Meir. Electrocardiographic and Autonomic Nervous System Changes after Changes in the Posture of Children and Adolescents with Duchenne Muscular Dystrophy. Arquivos brasileiros de cardiologia. vol 121. issue 2. 2024-04-10. PMID:38597534. |
duchenne muscular dystrophy (dmd) is a rare inherited neuromuscular disease. |
2024-04-10 |
2024-04-12 |
Not clear |