"Drisapersen"

Long-Term Efficacy, Safety, and Pharmacokinetics of Drisapersen in Duchenne Muscular Dystrophy: Results from an Open-Label Extension Study. Drisapersen induces exon 51 skipping during dystrophin pre-mRNA splicing and allows synthesis of partially functional dystrophin in Duchenne muscular dystrophy (DMD) patients with amenable mutations. This 188-week open-label extension of the dose-escalation study assessed the long-term efficacy, safety, and pharmacokinetics of drisapersen (PRO051/GSK2402968), 6 mg/kg subcutaneously, in 12 DMD subjects. Dosing was once weekly for 72 weeks. All subjects had a planned treatment interruption (weeks 73-80), followed by intermittent dosing (weeks 81-188). Subjects received a median (range) total dose of 5.93 (5.10 to 6.02) mg/kg drisapersen. After 177 weeks

Placebo‐controlled Phase 2 Trial of Drisapersen for Duchenne Muscular Dystrophy 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. Male DMD patients (≥5 years; time to rise from floor ≤15 s) were randomized to drisapersen 3 mg/kg/week, 6 mg/kg/week or placebo. The primary efficacy endpoint was change from baseline in 6-minute walking distance (6MWD) at week 24. Secondary endpoints included changes in timed function tests, muscle strength, and pulmonary function tests. Fifty-one patients were randomized to placebo ( = 16), drisapersen 3 mg/kg

A randomized placebo-controlled phase 3 trial of an antisense oligonucleotide, drisapersen, in Duchenne muscular dystrophy. 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. Drisapersen was generally well tolerated, with injection-site reactions and renal events as most commonly reported adverse events. A nonsignificant treatment difference (P = 0.415) in the change from baseline in six-minute walk distance (6MWD; primary efficacy endpoint) of 10.3 meters in favor of drisapersen was observed at week 48. Key secondary efficacy endpoints (North Star Ambulatory

GSK-2402968 (Drisapersen) for duchenne muscular dystrophy ? first line GSK-2402968 (Drisapersen) for duchenne muscular dystrophy – first line ..

Pharmacokinetics and safety of single doses of drisapersen in non-ambulant subjects with Duchenne muscular dystrophy: Results of a double-blind randomized clinical trial. Duchenne muscular dystrophy (DMD) is a progressive, lethal neuromuscular disorder caused by the absence of dystrophin protein due to mutations of the dystrophin gene. Drisapersen is a 2'-O-methyl-phosphorothioate oligonucleotide designed to skip exon 51 in dystrophin pre-mRNA to restore the reading frame of the mRNA. This study assessed safety, tolerability, and pharmacokinetics of drisapersen after a single subcutaneous administration in non-ambulatory subjects. Eligible subjects were non-ambulant boys aged ⩾9years, in wheelchairs for ⩾1 to ⩽4years, with a diagnosis of DMD resulting from a mutation correctable by drisapersen

Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, randomised, placebo-controlled phase 2 study. Duchenne muscular dystrophy is caused by dystrophin deficiency and muscle deterioration and preferentially affects boys. Antisense-oligonucleotide-induced exon skipping allows synthesis of partially functional dystrophin. We investigated the efficacy and safety of drisapersen, a 2'-O-methyl-phosphorothioate antisense oligonucleotide, given for 48 weeks. In this exploratory, double-blind, placebo-controlled study we recruited male patients (≥5 years of age; time to rise from floor ≤7 s) with Duchenne muscular dystrophy from 13 specialist centres in nine countries between Sept 1, 2010, and Sept 12, 2012. By use of a computer-generated

1 EMEA/H/C/003846 Questions and answers Withdrawal of the marketing authorisation application for Kyndrisa (drisapersen) On 31 May 2016, BioMarin International Limited officially notified the Committee for Medicinal Products for Human Use (CHMP) that it wishes to withdraw its application for a marketing authorisation for Kyndrisa, for the treatment of Duchenne muscular dystrophy. What is Kyndrisa? Kyndrisa is a medicine that contains the active substance drisapersen. It was to be available as a solution for injection under the skin. What was Kyndrisa expected to be used for? Kyndrisa was expected to be used for the treatment of Duchenne muscular dystrophy in patients aged 5 years and older who are able to walk and whose disease is due to certain genetic mutations (defects) that can

biomarker for Duchenne. Two natural history cohorts were studied including 168 longitudinal samples belonging to 66 patients. We further studied 1536 samples obtained from 3 independent clinical trials with drisapersen, an antisense oligonucleotide targeting exon 51: an open label study including 12 patients; a phase 3 randomized, double blind, placebo controlled study involving 186 patients; an open

Improving clinical trial design for Duchenne muscular dystrophy. Currently, the most promising therapies for Duchenne muscular dystrophy (DMD) are exon skipping and stop codon read-through, two strategies aimed at restoring the expression of dystrophin. A phase 3 clinical trial with drisapersen, a drug designed to induce exon 51-skipping, has failed to show significant improvement of the primary

Therapy” OR “Genome Editing” OR “Gene transfer therapy” OR “Exon skipping therapy” OR “CRISPR/Cas9” OR “CRISPR” OR “Delandistrogene moxeparvovec gene” OR “Adeno- associated virus” OR “AAV” OR (Micro dystrophin therapy” OR “Viltolarsen” OR “Eteplirsen” OR “Drisapersen” OR “Golodirsen” OR “casimersen” OR “Exon 53 skipping therapy ” OR “Exon 45 skipping therapy ” OR “Exon 51 skipping therapy ” OR “Cell

of the dystrophin gene to be skipped when it is transcribed to RNA for protein production, permitting a still-truncated but more functional version of the protein to be produced.[70] It is also known as nonsense suppression therapy.[71]Two kinds of antisense oligos, 2'-O-methyl phosphorothioate oligos (like drisapersen) and Morpholino oligos (like eteplirsen), have tentative evidence of benefit and are being

with drisapersen (PRO051) within the last 6 months. * Participation in any other DMD interventional clinical study within 12 weeks * Major change in physiotherapy regimen within the past 3 months * Major surgery within 3 months * Presence of other clinically significant illness * Use of an aminoglycoside antibiotic within 12 weeks or the need for this antibiotic or statin during study * Forced vital capacity

A Phase I/II, Open Label, Escalating Dose, Pilot Study to Assess Effect, Safety, Tolerability and PK of Multiple SC Doses of Drisapersen in Patients With Duchenne Muscular Dystrophy and to Assess the Potential for IV Dosing as an Alternative Route of Admi A Phase I/II, Open Label, Escalating Dose, Pilot Study to Assess Effect, Safety, Tolerability and PK of Multiple SC Doses of Drisapersen studies before adding more. A Phase I/II, Open Label, Escalating Dose, Pilot Study to Assess Effect, Safety, Tolerability and PK of Multiple SC Doses of Drisapersen in Patients With Duchenne Muscular Dystrophy and to Assess the Potential for IV Dosing as an Alternative Route of Administration The safety and scientific validity of this study is the responsibility of the study sponsor and investigators

within the past 14 weeks. * Received prior treatment with drisapersen. * Received any investigational drug within the past 3 months or 5 half-lives, whichever is longer.Contacts and LocationsGo to Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information Information from the National Library of Medicine To learn

) <40% of predicted value within 3 months of Screening or at the Screening visit * Known kidney disease or had an acute kidney injury within 6 months prior to Screening * Treatment with eteplirsen or drisapersen within 6 months prior to Screening, or any experimental gene therapy for the treatment of DMD at any time * Use of any herbal medication/supplement containing aristolochic acidOther inclusion

Drisapersen Duchenne Muscular Dystrophy (DMD) Treatment Protocol Drisapersen Duchenne Muscular Dystrophy (DMD) Treatment Protocol - Full Text View - ClinicalTrials.gov Try the modernized ClinicalTrials.gov beta website. Learn more about the modernization effort. Hide glossary GlossaryStudy record managers: refer to the Data Element Definitions if submitting registration or results information ).Please remove one or more studies before adding more. Drisapersen Duchenne Muscular Dystrophy (DMD) Treatment Protocol The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. ClinicalTrials.gov Identifier: NCT01890798

A Study of the Safety, Tolerability & Efficacy of Long-term Administration of Drisapersen in US & Canadian Subjects A Study of the Safety, Tolerability & Efficacy of Long-term Administration of Drisapersen in US & Canadian Subjects - Full Text View - ClinicalTrials.gov Try the modernized ClinicalTrials.gov beta website. Learn more about the modernization effort. Hide glossary GlossaryStudy record Save this study WarningYou have reached the maximum number of saved studies (100).Please remove one or more studies before adding more. A Study of the Safety, Tolerability & Efficacy of Long-term Administration of Drisapersen in US & Canadian Subjects The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has