Results 131 to 140 of about 71,938 (313)

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

Aggravation of Cardiovascular and Respiratory Decline in Advanced Duchenne Muscular Dystrophy Complicated by Dilated Cardiomyopathy – Case Study and Review of Literature

Journal of Education, Health and Sport
Introduction: Duchenne muscular dystrophy is a genetic X-linked recessive disorder. This condition is characterized by progressive loss of muscle tissue. Thus, it results in deterioration and inability to perform basic motor skills such as independent
Iwona Welian-Polus, Bartosz Mazur, Michał Bielak, Magdalena Mazur, Elżbieta Rypulak, Wiktoria Wilanowska, Anna Greguła, Karol Stachyrak, Dawid Mika, Kamila Turek   +9 more
doaj   +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

Efficacy and safety of Viltepso® in Duchenne muscular dystrophy: review of clinical studies

Русский журнал детской неврологии
The author presents a literature review on the safety and efficacy of antisense oligonucleotides in the treatment of Duchenne muscular dystrophy using the exon skipping method using Viltepso® (viltolarsen), the only drug of this class registered in ...
V. M. Suslov, D. I. Rudenko
doaj   +1 more source

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

Fast Myosin Binding Protein‐C Is a Vital Regulator in Young and Aged Fast Skeletal Muscle Homeostasis

Journal of Cachexia, Sarcopenia and Muscle, Volume 16, Issue 6, December 2025.
ABSTRACT Background Skeletal muscle plays a vital role in voluntary movement and locomotion. Fast‐twitch muscle fibres are characterized by their rapid contraction kinetics, high‐force generation and a distinct gene expression profile compared to slow‐twitch fibres.
Akhil Baby, Kalyani Ananthamohan, Brenna N. McIntyre, Sankar Natesan, Taejeong Song   +4 more
wiley   +1 more source

Carrier detection of duchenne and becker muscular dystrophy using muscle dystrophin immunohistochemistry [PDF]

, 1992
Acary Souza Bullé Oliveira, Alberto Alain Gabbai, Beny Schmidt, Beatriz Hitomi Kiyomoto, G. Camargo Lima, Carlo Minetti, Eduardo Bonilla   +6 more
openalex   +1 more source

Role of Peptides in Skeletal Muscle Wasting: A Scoping Review

Journal of Cachexia, Sarcopenia and Muscle, Volume 16, Issue 6, December 2025.
ABSTRACT Background Systemic muscle wasting is a prevalent condition that predicts adverse health outcomes in aging and disease. Despite its clinical relevance, the development of predictive biomarkers and effective pharmacological therapies remains limited. Peptides have recently gained attention for their diverse bioactive functions, positioning them
Petar Naumovski, Bart De Spiegeleer, Aster Wakjira, Christophe Van De Wiele, Vincent Mouly, Katarzyna Goljanek‐Whysall, Kauê Santana da Costa, Ewerton Cristhian Lima de Oliveira, Evelien Wynendaele, Anton De Spiegeleer   +9 more
wiley   +1 more source

Quantitative ultrasound assessment of Duchenne muscular dystrophy using edge detection analysis [PDF]

, 2016
Aarnink, Arai, Bhansing, Bickerstaffe, Boland, Bushby, Bushby, Canny, Chen, Emery, Gudmundsson, Hammoude, Hoffman, Hou, Jansen, Janssen, Mahmoud, Mazzone, Mazzone, McDonald, Peters, Pillen, Pillen, Rutkove, Schreiber, Shklyar, Torriani, Wu, Zaidman, Zaidman, Zwehl   +30 more
core   +2 more sources

Descriptive Phenotype of Obsessive Compulsive Symptoms in Males With Duchenne Muscular Dystrophy

, 2018
Angela Lee, Edward T. Buckingham, Aaron J. Kauer, Katherine D. Mathews   +3 more
openalex   +2 more sources