Results 181 to 190 of about 114,439 (345)

573. Enhanced Level of Gene Correction Mediated by Oligonucleotides Containing CpG Modification in the mdx Mouse Model for Duchenne Muscular Dystrophy [PDF]

, 2006
Carmen Bertoni, Arjun Rustagi, Thomas A. Rando   +2 more
openalex   +1 more source

A Fast Path from Innovation to Safe and Effective Medicines

Clinical Pharmacology &Therapeutics, Volume 118, Issue 6, Page 1392-1404, December 2025.
Consistent progress in medicines development has allowed both for de novo treatment options and for the refinement of existing products that improve effectiveness or reduce harm. Nonetheless, unmet medical needs persist, particularly in rare diseases, pediatrics and underserved populations.
Peter Arlett, Falk Ehmann, Ralf Herold, Evdokia Korakianiti, Pierpaolo Moscariello, Liese Barbier, Iordanis Gravanis, Frank Pétavy, Emmanuel Cormier, Andrew Thomson, Spiros Vamvakas, Steffen Thirstrup   +11 more
wiley   +1 more source

Publisher Correction: Modelling Duchenne muscular dystrophy in MYOD1-converted urine-derived cells treated with 3-deazaneplanocin A hydrochloride [PDF]

, 2020
Hotake Takizawa, Yuko Hara–Azumi, Yoshitaka Mizobe, Taisuke Ohno, Sadafumi Suzuki, Ken Inoue, Eri Takeshita, Yuko Shimizu‐Motohashi, Akihiko Ishiyama, Mikio Hoshino, Hirofumi Komaki, Shin’ichi Takeda, Yoshitsugu Aoki   +12 more
openalex   +1 more source

Nutritional status, swallowing disorders, and respiratory prognosis in adult Duchenne muscular dystrophy patients [PDF]

, 2021
Abdallah Fayssoil, Cendrine Chaffaut, Hélène Prigent, Pascal Laforêt, Bernard Clair, David Orlikowski, Adam Ogna, Sylvie Chevret, Paris Meng, Djillali Annane, Frédéric Lofaso, Pascal Crenn   +11 more
openalex   +1 more source

Antisense oligonucleotides targeting valosin‐containing protein ameliorate muscle pathology and molecular defects in cell and mouse models of multisystem proteinopathy

Clinical and Translational Medicine, Volume 15, Issue 12, December 2025.
Multisystem proteinopathy 1 (MSP1), caused by gain‐of‐function VCP variants, leads to multisystem degeneration. Using VCP patient‐derived hiPSCs, skeletal muscle progenitor cells were generated to evaluate antisense oligonucleotide (ASO) therapy.
Pallabi Pal, Michele Carrer, Lan Weiss, Olga G. Jaime, Cheng Cheng, Alyaa Shmara, Victoria Boock, Danae Bosch, Marwan Youssef, Yasamin Fazeli, Megan Afetian, Tamar R. Grossman, Michael R. Hicks, Paymaan Jafar‐nejad, Virginia Kimonis   +14 more
wiley   +1 more source

Non-operative treatment for perforated gastro-duodenal peptic ulcer in Duchenne Muscular Dystrophy: a case report

BMC Surgery, 2004
Background Clinical characteristics and complications of Duchenne muscular dystrophy caused by skeletal and cardiac muscle degeneration are well known. Gastro-intestinal involvement has also been recognised in these patients.
Wever Jan, Van Royen Barend J, Oddens Jorg R, Brinkman Justus-Martijn, Olsman Jan G   +4 more
doaj  

Smiling is a Costly Signal of Cooperation Opportunities: Experimental Evidence from a Trust Game [PDF]

We test the hypothesis that "genuine" or "convincing" smiling is a costly signal that has evolved to induce cooperation in situations requiring mutual trust. Potential trustees in a trust game made video clips for viewing by potential trusters before the
Centorrino, Samuele, Djemaï, Elodie, Hopfensitz, Astrid, Milinski, Manfred, Seabright, Paul   +4 more
core  

PRDX5 Regulates Mitochondrial Function and Nuclear Spreading in Myogenesis and Acts With PRDX3 to Delay Muscle Aging

Journal of Cachexia, Sarcopenia and Muscle, Volume 16, Issue 6, December 2025.
ABSTRACT Background Skeletal muscle aging is associated with oxidative stress and mitochondrial dysfunction. Peroxiredoxins (PRDXs), particularly PRDX3 and PRDX5, are antioxidant enzymes that are uniquely localized to mitochondria. While PRDX3 has been reported to play a role in maintaining mitochondrial function in muscle, the specific function of ...
Joonho Suh, Je‐Hyun Eom, Jongmin Baik, Wonn Shim, Max A. Tischfield, Hyun Ae Woo, Yun‐Sil Lee   +6 more
wiley   +1 more source

Diagnosis and management of Duchenne muscular dystrophy, part 2: respiratory, cardiac, bone health, and orthopaedic management

Lancet Neurology, 2018
D. Birnkrant, K. Bushby, C. Bann, B. Alman, S. Apkon, Angela Blackwell, L. Case, L. Cripe, S. Hadjiyannakis, A. Olson, Daniel W. Sheehan, J. Bolen, D. Weber, L. Ward   +13 more
semanticscholar   +1 more source

Somatic gene editing ameliorates skeletal and cardiac muscle failure in pig and human models of Duchenne muscular dystrophy

Nature Network Boston, 2020
A. Moretti, LM Fonteyne, F. Giesert, P. Hoppmann, A. Meier, T. Bozoglu, A. Baehr, C. Schneider, D. Sinnecker, K. Klett, T. Fröhlich, F. Rahman, T. Haufe, S. Sun, V. Jurisch, B. Kessler, R. Hinkel, R. Dirschinger, Eimo Martens, C. Jilek, A. Graf, S. Krebs, Gianluca Santamaria, M. Kurome, V. Zakhartchenko, B. Campbell, K. Voelse, A. Wolf, Tilman Ziegler, S. Reichert, Seungmin Lee, F. Flenkenthaler, Tatjana Dorn, I. Jeremias, H. Blum, A. Dendorfer, A. Schnieke, S. Krause, M. Walter, N. Klymiuk, K. Laugwitz, E. Wolf, W. Wurst, W. Wurst, C. Kupatt   +44 more
semanticscholar   +1 more source