[HTML][HTML] AMONDYS 45 (Casimersen), a Novel Antisense Phosphorodiamidate Morpholino Oligomer: Clinical Considerations for Treatment in Duchenne Muscular …
ME Vasterling, RJ Maitski, BA Davis, JE Barnes… - Cureus, 2023 - ncbi.nlm.nih.gov
AMONDYS 45 (casimersen) is an antisense oligonucleotide therapy used to treat Duchenne
muscular dystrophy (DMD), a rare genetic disorder characterized by a mutation in the DMD …
muscular dystrophy (DMD), a rare genetic disorder characterized by a mutation in the DMD …
Pilot investigations into the mechanistic basis for adverse effects of glucocorticoids in dysferlinopathy
Background Dysferlinopathies are a clinically heterogeneous group of muscular dystrophies
caused by gene mutations resulting in deficiency of the membrane-associated protein …
caused by gene mutations resulting in deficiency of the membrane-associated protein …
The BALB/c. mdx62 mouse exhibits a dystrophic muscle pathology and is a model of Duchenne muscular dystrophy
K Swiderski, AS Chan, MJ Herold… - Disease Models & …, 2024 - journals.biologists.com
Duchenne muscular dystrophy (DMD) is a devastating monogenic skeletal muscle-wasting
disorder. Although many pharmacological and genetic interventions have been reported in …
disorder. Although many pharmacological and genetic interventions have been reported in …