FRINK Linked Data Fragments server

Matches in SemOpenAlex for { <https://semopenalex.org/work/W4367304850> ?p ?o ?g. }

• W4367304850 abstract "<h3>Objective:</h3> To assess performance of upper limb (PUL) function in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD) receiving ataluren plus standard of care (SoC) in Study 041 (NCT03179631) and in the STRIDE Registry (NCT02369731). <h3>Background:</h3> DMD is a severe neuromuscular disorder caused by a lack of functional dystrophin. Ataluren promotes read through of an in-frame premature stop codon to produce full-length dystrophin and is indicated for the treatment of patients with nmDMD. Study 041 (NCT03179631) is a phase 3, double-blind, placebo-controlled 72-week trial. The STRIDE Registry (NCT02369731) is an ongoing, long-term, real-world evidence study. <h3>Design/Methods:</h3> In Study 041, boys with nmDMD aged ≥5 years, on a stable corticosteroid regimen, and with a 6-minute walk distance (6MWD) ≥150m were randomized 1:1, ataluren:placebo. The intention-to-treat (ITT) population comprised randomized boys who received at least one dose of study treatment (N=359; mean age 8.1 years); a key subgroup included those with baseline 300–400m 6MWD (n=169). STRIDE patients were propensity-score matched to patients receiving SoC alone in CINRG DNHS (NCT00468832), yielding a comparable population (N=261). Kaplan-Meier analyses estimated age at loss of upper limb function. <h3>Results:</h3> Least-squares mean PUL total score change from baseline to week 72 (by MMRM analysis) numerically favored ataluren versus placebo (0.44, <i>p</i>=0.1059) in the Study 041 ITT population and was significant in the 300–400m 6MWD subgroup (1.02, <i>p</i>=0.0165). In matched STRIDE versus CINRG patients (mean last assessment age, 13.1 versus 14.6), ataluren preserved hand-to-mouth function by 3.4 years (<i>p</i>=0.0046) as assessed by entry level items of PUL versus Brooke Scale, respectively. Median age at loss of overhead reach numerically favored STRIDE, consistent with the overall trend (15.8 versus 12.6; <i>p</i>=0.2872). Median age at loss of distal hand function was non-estimable for STRIDE patients. <h3>Conclusions:</h3> These results indicate that ataluren may help preserve upper limb function in patients with advanced nmDMD. <b>Disclosure:</b> Dr. McDonald has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Sarepta Therapeutics. Dr. McDonald has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for PTC Therapeutics. Dr. McDonald has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Astellas. Dr. McDonald has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Capricor Therapeutics. Dr. McDonald has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Catabasis. Dr. McDonald has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Epirium Bio. Dr. McDonald has received personal compensation in the range of $0-$499 for serving as a Consultant for Italfarmaco. Dr. McDonald has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Santhera Pharmaceuticals. Dr. McDonald has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sarepta Therapeutics. Dr. McDonald has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for PTC Therapeutics. Dr. McDonald has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Capricor. Dr. McDonald has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Catabasis Pharmaceuticals. Dr. McDonald has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Santhera Pharmaceuticals. The institution of Dr. McDonald has received research support from Sarepta Therapeutics. The institution of Dr. McDonald has received research support from PTC Therapeutics. The institution of Dr. McDonald has received research support from Santhera Pharmaceuticals. The institution of Dr. McDonald has received research support from Astellas. The institution of Dr. McDonald has received research support from Capricor Therapeutics. The institution of Dr. McDonald has received research support from Catabasis. The institution of Dr. McDonald has received research support from Epirium Bio. The institution of Dr. McDonald has received research support from Italfarmaco. Dr. McDonald has received research support from NS Pharma. The institution of Dr. McDonald has received research support from Pfizer. The institution of Dr. McDonald has received research support from NIH (NINDS). The institution of Dr. McDonald has received research support from NIDILRR. The institution of Dr. McDonald has received research support from Department of Defense. The institution of Dr. McDonald has received research support from Parent Project Muscular Dystrophy. The institution of Dr. McDonald has received research support from Muscular Dystrophy Association. Dr. McDonald has received personal compensation in the range of $500-$4,999 for serving as a Member National Advisory Board for Medical Rehabilitation Research with NIH. Eugenio Mercuri has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sarepta, Santhera, PTC, Pfizer, Roche, Biogen, Avexis Novartis, Scholar Rock, Cytokinetics. Eugenio Mercuri has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Sarepta, Santhera, PTC, Pfizer, Roche, Biogen, Avexis Novartis. The institution of Eugenio Mercuri has received research support from biogen. Dr. Muntoni has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sarepta. Dr. Muntoni has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Pfizer. Dr. Muntoni has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Sarepta. Dr. Muntoni has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Pfizer. Dr. Muntoni has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Biogen. Dr. Muntoni has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Avexis. Dr. Muntoni has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Roche. The institution of Dr. Muntoni has received research support from European Commission. The institution of Dr. Muntoni has received research support from Medical Research Council. The institution of Dr. Muntoni has received research support from Biogen. The institution of Dr. Muntoni has received research support from Muscular Dystrophy UK. The institution of Dr. Muntoni has received research support from MDA USA. The institution of Dr. Muntoni has received research support from Sarepta. The institution of Dr. Muntoni has received research support from Association Francoise Myopathies. Dr. Muntoni has received personal compensation in the range of $0-$499 for serving as a Clinical expert with UK NICE Committee. Heather Gordish-Dressman has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for AGADA BioSciences. Heather Gordish-Dressman has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for TRiNDS, LLC. Heather Gordish-Dressman has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Solid Biosciences. An immediate family member of Heather Gordish-Dressman has received intellectual property interests from a discovery or technology relating to health care. Ms. Morgenroth has received personal compensation for serving as an employee of TRiNDS. Ms. Morgenroth has stock in TRiNDS. Maria Bernadete Dutra de Resende has nothing to disclose. Shuizhen Zhou has nothing to disclose. Dr. Neeharika has nothing to disclose. Dr. Haginoya has nothing to disclose. Dr. Ramos-Platt has received personal compensation for serving as an employee of MDA. Dr. Ramos-Platt has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Guidepoint. The institution of Dr. Ramos-Platt has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Biogen. Dr. Ramos-Platt has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Cure sma. Mrs. Williams has nothing to disclose. Vinay Penematsa has nothing to disclose. Connie Chou has stock in PTC Therapeutics. Dr. Lin has received personal compensation for serving as an employee of PTC Therapeutics. Ms. Johnson has received personal compensation for serving as an employee of PTC Therapeutics Inc. Ms. Johnson has stock in PTC Therapeutics. Dr. Koladicz has received personal compensation for serving as an employee of PTC Therapeutics. Nick Mastrandrea has received personal compensation for serving as an employee of PTC Therapeutics. Nick Mastrandrea has stock in PTC Therapeutics. Christian Werner has nothing to disclose. Panayiota Trifillis has received personal compensation for serving as an employee of PTC Therapeutics. Panayiota Trifillis has received stock or an ownership interest from PTC Therapeutics, Inc.. Panayiota Trifillis has received intellectual property interests from a discovery or technology relating to health care." @default.
• W4367304850 created "2023-04-29" @default.
• W4367304850 creator A5005994885 @default.
• W4367304850 creator A5008253139 @default.
• W4367304850 creator A5008532884 @default.
• W4367304850 creator A5015920322 @default.
• W4367304850 creator A5018100580 @default.
• W4367304850 creator A5037277344 @default.
• W4367304850 creator A5037835941 @default.
• W4367304850 creator A5040696668 @default.
• W4367304850 creator A5042269980 @default.
• W4367304850 creator A5043186708 @default.
• W4367304850 creator A5045097830 @default.
• W4367304850 creator A5051207729 @default.
• W4367304850 creator A5060575192 @default.
• W4367304850 creator A5071163954 @default.
• W4367304850 creator A5071684535 @default.
• W4367304850 creator A5077421484 @default.
• W4367304850 creator A5079323039 @default.
• W4367304850 creator A5082896865 @default.
• W4367304850 creator A5089234882 @default.
• W4367304850 date "2023-04-25" @default.
• W4367304850 modified "2023-09-26" @default.
• W4367304850 title "Ataluren Preserves Upper Limb Function in nmDMD Patients from Study 041, a Phase 3 Placebo-Controlled Trial, and the STRIDE Registry (S34.008)" @default.
• W4367304850 doi "https://doi.org/10.1212/wnl.0000000000202507" @default.
• W4367304850 hasPublicationYear "2023" @default.
• W4367304850 type Work @default.
• W4367304850 citedByCount "0" @default.
• W4367304850 crossrefType "proceedings-article" @default.
• W4367304850 hasAuthorship W4367304850A5005994885 @default.
• W4367304850 hasAuthorship W4367304850A5008253139 @default.
• W4367304850 hasAuthorship W4367304850A5008532884 @default.
• W4367304850 hasAuthorship W4367304850A5015920322 @default.
• W4367304850 hasAuthorship W4367304850A5018100580 @default.
• W4367304850 hasAuthorship W4367304850A5037277344 @default.
• W4367304850 hasAuthorship W4367304850A5037835941 @default.
• W4367304850 hasAuthorship W4367304850A5040696668 @default.
• W4367304850 hasAuthorship W4367304850A5042269980 @default.
• W4367304850 hasAuthorship W4367304850A5043186708 @default.
• W4367304850 hasAuthorship W4367304850A5045097830 @default.
• W4367304850 hasAuthorship W4367304850A5051207729 @default.
• W4367304850 hasAuthorship W4367304850A5060575192 @default.
• W4367304850 hasAuthorship W4367304850A5071163954 @default.
• W4367304850 hasAuthorship W4367304850A5071684535 @default.
• W4367304850 hasAuthorship W4367304850A5077421484 @default.
• W4367304850 hasAuthorship W4367304850A5079323039 @default.
• W4367304850 hasAuthorship W4367304850A5082896865 @default.
• W4367304850 hasAuthorship W4367304850A5089234882 @default.
• W4367304850 hasConcept C121332964 @default.
• W4367304850 hasConcept C142724271 @default.
• W4367304850 hasConcept C18007350 @default.
• W4367304850 hasConcept C1862650 @default.
• W4367304850 hasConcept C204787440 @default.
• W4367304850 hasConcept C27081682 @default.
• W4367304850 hasConcept C41008148 @default.
• W4367304850 hasConcept C44280652 @default.
• W4367304850 hasConcept C62520636 @default.
• W4367304850 hasConcept C71924100 @default.
• W4367304850 hasConcept C99508421 @default.
• W4367304850 hasConceptScore W4367304850C121332964 @default.
• W4367304850 hasConceptScore W4367304850C142724271 @default.
• W4367304850 hasConceptScore W4367304850C18007350 @default.
• W4367304850 hasConceptScore W4367304850C1862650 @default.
• W4367304850 hasConceptScore W4367304850C204787440 @default.
• W4367304850 hasConceptScore W4367304850C27081682 @default.
• W4367304850 hasConceptScore W4367304850C41008148 @default.
• W4367304850 hasConceptScore W4367304850C44280652 @default.
• W4367304850 hasConceptScore W4367304850C62520636 @default.
• W4367304850 hasConceptScore W4367304850C71924100 @default.
• W4367304850 hasConceptScore W4367304850C99508421 @default.
• W4367304850 hasLocation W43673048501 @default.
• W4367304850 hasOpenAccess W4367304850 @default.
• W4367304850 hasPrimaryLocation W43673048501 @default.
• W4367304850 hasRelatedWork W1978824385 @default.
• W4367304850 hasRelatedWork W2009832852 @default.
• W4367304850 hasRelatedWork W2013840730 @default.
• W4367304850 hasRelatedWork W2031351879 @default.
• W4367304850 hasRelatedWork W2136182926 @default.
• W4367304850 hasRelatedWork W2329167787 @default.
• W4367304850 hasRelatedWork W2600394844 @default.
• W4367304850 hasRelatedWork W2748952813 @default.
• W4367304850 hasRelatedWork W2899084033 @default.
• W4367304850 hasRelatedWork W4233016836 @default.
• W4367304850 isParatext "false" @default.
• W4367304850 isRetracted "false" @default.
• W4367304850 workType "article" @default.