Exon skipping therapy for Duchenne muscular dystrophy

Advanced Drug Delivery Reviews

Volumn 87, Issue , 2015, Pages 104-107

  Kole, Ryszard ^a   Krieg, Arthur M ^b  

^a AVI BIOPHARMA INC   (United States)

^b Checkmate Pharmaceuticals   (United States)

Author keywords

Clinical trials; Drisapersen; Eteplirsen; Exon skipping; RNA splicing; RNA therapeutics

Indexed keywords

CYTOLOGY; DETERIORATION; MUSCLE; OLIGONUCLEOTIDES; RNA;

CLINICAL TRIAL; DRISAPERSEN; ETEPLIRSEN; EXON SKIPPING; RNA SPLICING; RNA THERAPEUTICS;

NEUROMUSCULAR REHABILITATION;

DRISAPERSEN; DYSTROPHIN; ETEPLIRSEN; PLACEBO; AVI-4658; OLIGONUCLEOTIDE;

CHEMICAL COMPOSITION; CHEMICAL STRUCTURE; DRUG EFFICACY; DRUG HYPERSENSITIVITY; DRUG RESPONSE; DRUG SAFETY; DUCHENNE MUSCULAR DYSTROPHY; EXON SKIPPING; FLU LIKE SYNDROME; HUMAN; INFUSION RELATED REACTION; INJECTION SITE DISCOLORATION; INJECTION SITE ERYTHEMA; INJECTION SITE INDURATION; INJECTION SITE PAIN; INJECTION SITE REACTION; LUNG FUNCTION; NEPHRITIS; NONHUMAN; PHASE 2 CLINICAL TRIAL (TOPIC); PHASE 3 CLINICAL TRIAL (TOPIC); PRIORITY JOURNAL; PROTEINURIA; RANDOMIZED CONTROLLED TRIAL (TOPIC); REVIEW; UNSPECIFIED SIDE EFFECT; WALKING; ANIMAL; BIOSYNTHESIS; CHEMISTRY; DRUG EFFECTS; EXON; GENETICS; MUSCULAR DYSTROPHY, DUCHENNE; MUTATION; RNA SPLICING;

ANIMALS; DYSTROPHIN; EXONS; HUMANS; MUSCULAR DYSTROPHY, DUCHENNE; MUTATION; OLIGONUCLEOTIDES; RANDOMIZED CONTROLLED TRIALS AS TOPIC; RNA SPLICING;

EID: 84937251741     PISSN: 0169409X     EISSN: 18728294     Source Type: Journal    
DOI: 10.1016/j.addr.2015.05.008     Document Type: Review

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