PRO051

is a synthetic, modified RNA molecule; Intramuscular injection of antisense oligonucleotide PRO051 induced dystrophin synthesis in four patients with Duchenne's muscular dystrophy who had suitable mutations

Also Known As:

Networked: 17 relevant articles (2 outcomes, 7 trials/studies)

Relationship Network

Bio-Agent Context: Research Results

Experts

1.	Campion, Giles: 6 articles (08/2018 - 01/2014)
2.	Lourbakos, Afrodite: 4 articles (08/2018 - 10/2014)
3.	Goemans, Nathalie: 3 articles (02/2019 - 03/2017)
4.	Wilson, Rosamund: 3 articles (08/2018 - 10/2014)
5.	Kraus, John E: 3 articles (01/2018 - 01/2014)
6.	Goemans, Nathalie M: 3 articles (01/2016 - 12/2007)
7.	de Kimpe, Sjef J: 3 articles (01/2016 - 12/2007)
8.	van Deutekom, Judith C: 3 articles (01/2016 - 12/2007)
9.	Campbell, Craig: 2 articles (01/2020 - 01/2018)
10.	Flanigan, Kevin M: 2 articles (08/2018 - 01/2014)

Related Diseases

1.	Duchenne Muscular Dystrophy (Muscular Dystrophy, Becker) 10/01/2014 - "Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, randomised, placebo-controlled phase 2 study." 08/01/2018 - "This double-blind, randomized, placebo-controlled Phase 2 study (NCT01462292) assessed the 24-week efficacy, safety, tolerability, and pharmacokinetics of two different subcutaneous drisapersen doses, and the 24-week off-dose persistent effect, in ambulant Duchenne muscular dystrophy (DMD) patients. " 08/01/2018 - "Placebo-controlled Phase 2 Trial of Drisapersen for Duchenne Muscular Dystrophy." 01/01/2018 - "This 48-week, randomized, placebo-controlled phase 3 study (DMD114044; NCT01254019) evaluated efficacy and safety of subcutaneous drisapersen 6 mg/kg/week in 186 ambulant boys aged ≥5 years, with Duchenne muscular dystrophy (DMD) resulting from an exon 51 skipping amenable mutation. " 01/01/2016 - "Long-Term Efficacy, Safety, and Pharmacokinetics of Drisapersen in Duchenne Muscular Dystrophy: Results from an Open-Label Extension Study."
2.	Muscular Dystrophies (Muscular Dystrophy) 04/21/2011 - "Systemically administered PRO051 showed dose-dependent molecular efficacy in patients with Duchenne's muscular dystrophy, with a modest improvement in the 6-minute walk test after 12 weeks of extended treatment. " 04/21/2011 - "Systemic administration of PRO051 in Duchenne's muscular dystrophy." 12/27/2007 - "Intramuscular injection of antisense oligonucleotide PRO051 induced dystrophin synthesis in four patients with Duchenne's muscular dystrophy who had suitable mutations, suggesting that further studies might be feasible." 04/21/2011 - "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. "
3.	Injection Site Reaction 02/01/2019 - "Injection site reactions after long-term subcutaneous delivery of drisapersen: a retrospective study." 01/01/2019 - "Magnetic resonance imaging characteristics of injection site reactions after long-term subcutaneous delivery of drisapersen." 01/01/2018 - "However, drisapersen resulted in a significant increase in injection site reactions (risk ratio [RR] 3.67, 95% CI 1.96 to 6.89, p < 0.0001) and renal toxicity (RR 1.81, 95% CI 1.11 to 2.94, p = 0.02). " 01/01/2018 - "Drisapersen was generally well tolerated, with injection-site reactions and renal events as most commonly reported adverse events. " 08/01/2018 - "Most common adverse events in both drisapersen groups were related to injection site reactions and subclinical proteinuria. "
4.	Disease Progression 03/01/2017 - "Comparison of ambulatory capacity and disease progression of Duchenne muscular dystrophy subjects enrolled in the drisapersen DMD114673 study with a matched natural history cohort of subjects on daily corticosteroids." 01/01/2022 - "We identified a total of 23 prognostic indicators of disease progression in DMD, namely age at diagnosis, age at onset of symptoms, ataluren treatment, ATL1102, BMI, cardiac medication, DMD genetic modifiers, DMD mutation type, drisapersen, edasalonexent, eteplirsen, glucocorticoid exposure, height, idebenone, lower limb surgery, orthoses, oxandrolone, spinal surgery, TAS-205, vamorolone, vitlolarsen, ventilation support, and weight. "
5.	Proteinuria 08/01/2018 - "Most common adverse events in both drisapersen groups were related to injection site reactions and subclinical proteinuria. " 10/01/2014 - "The most common adverse events in drisapersen-treated patients were injection-site reactions (14 patients given continuous drisapersen, 15 patients given intermittent drisapersen, and six given placebo) and renal events (13 for continuous drisapersen, 12 for intermittent drisapersen, and seven for placebo), most of which were subclinical proteinuria. "

Related Drugs and Biologics

1.	Antisense Oligonucleotides
2.	Dystrophin
3.	Antisense RNA (Anti-Sense RNA)
4.	Oligonucleotides
5.	Adrenal Cortex Hormones (Corticosteroids)
6.	eteplirsen
7.	Chemokine CCL2 (Monocyte Chemoattractant Protein 1)
8.	Cytokines
9.	Messenger RNA (mRNA)
10.	RNA Precursors (Precursor, mRNA)

Related Therapies and Procedures

1.	Intramuscular Injections
2.	Therapeutics
3.	Orthotic Devices (Orthosis)
4.	Subcutaneous Injections