Systemic administration of PRO051 in Duchenne's muscular dystrophy.

• Local intramuscular administration of the antisense oligonucleotide PRO051 in patients with Duchenne's muscular dystrophy with relevant mutations was previously reported to induce the skipping of exon 51 during pre-messenger RNA splicing of the dystrophin gene and to facilitate new dystrophin expression in muscle-fiber membranes. The present phase 1-2a study aimed to assess the safety, pharmacokinetics, and molecular and clinical effects of systemically administered PRO051.

Nyckelord

Adolescent

Alternative Splicing

Child

Child

Preschool

Creatine Kinase

urine

Dose-Response Relationship

Drug

Dystrophin

genetics

metabolism

Exercise Test

Exons

Humans

Injections

Subcutaneous

Male

Muscle Strength

drug effects

Muscle

Skeletal

chemistry

metabolism

Muscular Dystrophy

Duchenne

drug therapy

genetics

Mutation

Oligonucleotides

administration & dosage

adverse effects

blood

therapeutic use

RNA

analysis

Publikations- och innehållstyp

vet (ämneskategori)

art (ämneskategori)