Safety and efficacy of viltolarsen in ambulatory and nonambulatory males with Duchenne muscular dystrophy

Abstract Duchenne muscular dystrophy (DMD) is an X-linked recessive disease characterized by mutations in the dystrophin gene, causing motor and pulmonary function decline. Viltolarsen is indicated for patients with dystrophin gene mutations amenable to exon 53 skipping. Here, we report safety, moto...

Whakaahuatanga katoa

Ngā taipitopito rārangi puna kōrero

Ngā kaituhi matua:	Amy D. Harper, Haluk Topaloglu, Eugenio Mercuri, Vasiliy Suslov, Liwen Wu, Cigdem Y. Ayanoglu, Michael Tansey, Michelle L. Previtera, Robert A. Crozier, Leslie Magnus, Paula R. Clemens
Hōputu:	Artigo
Reo:	Inglês
I whakaputaina:	Nature Portfolio 2024-10-01
Rangatū:	Scientific Reports
Ngā marau:	Neuromuscular disease Pediatric research
Urunga tuihono:	https://doi.org/10.1038/s41598-024-70783-y
Ngā Tūtohu:	Tāpirihia he Tūtohu Kāore He Tūtohu, Me noho koe te mea tuatahi ki te tūtohu i tēnei pūkete!