FRINK Linked Data Fragments server

Matches in SemOpenAlex for { <https://semopenalex.org/work/W4387563853> ?p ?o ?g. }

• W4387563853 abstract "To assess compound muscle action potential (CMAP) amplitudes as electrophysiologic markers in relation to clinical outcome in adult patients with 5q-linked spinal muscular atrophy (SMA) before and during treatment with risdiplam.In this monocentric longitudinal cohort study, CMAP of 18 adult patients with SMA type 2 or 3 were assessed at baseline (T0 ) and after 10 months (T10 ) of risdiplam treatment. CMAP amplitudes of the median, ulnar, peroneal, and tibial nerves were compared with established clinical outcome scores, and with the course of disease before start of treatment.During a pharmacotherapy-naive pre-treatment period of 328 ± 46 days, Revised Upper Limb Module (RULM) score and peroneal nerve CMAP amplitudes decreased, while CMAP of tibial and upper limb nerves remained unchanged. CMAP amplitudes positively correlated with clinical scores (Hammersmith Functional Motor Scale-Expanded [HFMSE], RULM) at T0 . During risdiplam treatment, HFMSE and Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) scores increased, paralleled by marked increase of CMAP amplitudes in both median nerves (T10 -T0 ; right: Δ = 1.4 ± 1.4 mV, p = 0.0003; left: Δ = 1.3 ± 1.4 mV, p = 0.0007), but not in ulnar, peroneal, or tibial nerves. A robust increase of median nerve CMAP amplitudes correlated well with an increase in the HFMSE score (T10 -T0 ). Median nerve CMAP amplitudes at T0 were associated with subsequent risdiplam-related improvement of HFMSE and CHOP INTEND scores at T10 .Median nerve CMAP amplitudes increase with risdiplam treatment in adult SMA patients, and should be further evaluated as potential easy-to-use electrophysiologic markers in assessing and monitoring clinical response to therapy." @default.
• W4387563853 created "2023-10-13" @default.
• W4387563853 creator A5043733377 @default.
• W4387563853 creator A5052487829 @default.
• W4387563853 creator A5080482957 @default.
• W4387563853 creator A5090306990 @default.
• W4387563853 date "2023-10-12" @default.
• W4387563853 modified "2023-10-13" @default.
• W4387563853 title "Evaluation of risdiplam efficacy in 5q spinal muscular atrophy: A systematic comparison of electrophysiologic with clinical outcome measures" @default.
• W4387563853 cites W1963604250 @default.
• W4387563853 cites W1980477780 @default.
• W4387563853 cites W1992974743 @default.
• W4387563853 cites W2046864260 @default.
• W4387563853 cites W2124852855 @default.
• W4387563853 cites W2125953041 @default.
• W4387563853 cites W2129290719 @default.
• W4387563853 cites W2150501546 @default.
• W4387563853 cites W2528264181 @default.
• W4387563853 cites W2671097371 @default.
• W4387563853 cites W2769295220 @default.
• W4387563853 cites W2770254801 @default.
• W4387563853 cites W2901782370 @default.
• W4387563853 cites W2910420119 @default.
• W4387563853 cites W2960407661 @default.
• W4387563853 cites W2966208621 @default.
• W4387563853 cites W3011851030 @default.
• W4387563853 cites W3032702744 @default.
• W4387563853 cites W3085019865 @default.
• W4387563853 cites W3094366038 @default.
• W4387563853 cites W3119705164 @default.
• W4387563853 cites W3123519011 @default.
• W4387563853 cites W3133270341 @default.
• W4387563853 cites W3175696934 @default.
• W4387563853 cites W3181959214 @default.
• W4387563853 cites W3186720955 @default.
• W4387563853 cites W3196123322 @default.
• W4387563853 cites W4200042237 @default.
• W4387563853 cites W4285795569 @default.
• W4387563853 cites W4298121256 @default.
• W4387563853 cites W4309151969 @default.
• W4387563853 cites W4321612068 @default.
• W4387563853 cites W4366828999 @default.
• W4387563853 cites W4379769452 @default.
• W4387563853 doi "https://doi.org/10.1111/ene.16099" @default.
• W4387563853 hasPubMedId "https://pubmed.ncbi.nlm.nih.gov/37823715" @default.
• W4387563853 hasPublicationYear "2023" @default.
• W4387563853 type Work @default.
• W4387563853 citedByCount "0" @default.
• W4387563853 crossrefType "journal-article" @default.
• W4387563853 hasAuthorship W4387563853A5043733377 @default.
• W4387563853 hasAuthorship W4387563853A5052487829 @default.
• W4387563853 hasAuthorship W4387563853A5080482957 @default.
• W4387563853 hasAuthorship W4387563853A5090306990 @default.
• W4387563853 hasBestOaLocation W43875638531 @default.
• W4387563853 hasConcept C114614502 @default.
• W4387563853 hasConcept C126322002 @default.
• W4387563853 hasConcept C151626666 @default.
• W4387563853 hasConcept C161921814 @default.
• W4387563853 hasConcept C164705383 @default.
• W4387563853 hasConcept C185263204 @default.
• W4387563853 hasConcept C24998067 @default.
• W4387563853 hasConcept C2776825438 @default.
• W4387563853 hasConcept C2778558090 @default.
• W4387563853 hasConcept C2779134260 @default.
• W4387563853 hasConcept C33923547 @default.
• W4387563853 hasConcept C42219234 @default.
• W4387563853 hasConcept C71924100 @default.
• W4387563853 hasConceptScore W4387563853C114614502 @default.
• W4387563853 hasConceptScore W4387563853C126322002 @default.
• W4387563853 hasConceptScore W4387563853C151626666 @default.
• W4387563853 hasConceptScore W4387563853C161921814 @default.
• W4387563853 hasConceptScore W4387563853C164705383 @default.
• W4387563853 hasConceptScore W4387563853C185263204 @default.
• W4387563853 hasConceptScore W4387563853C24998067 @default.
• W4387563853 hasConceptScore W4387563853C2776825438 @default.
• W4387563853 hasConceptScore W4387563853C2778558090 @default.
• W4387563853 hasConceptScore W4387563853C2779134260 @default.
• W4387563853 hasConceptScore W4387563853C33923547 @default.
• W4387563853 hasConceptScore W4387563853C42219234 @default.
• W4387563853 hasConceptScore W4387563853C71924100 @default.
• W4387563853 hasLocation W43875638531 @default.
• W4387563853 hasLocation W43875638532 @default.
• W4387563853 hasOpenAccess W4387563853 @default.
• W4387563853 hasPrimaryLocation W43875638531 @default.
• W4387563853 hasRelatedWork W1493579730 @default.
• W4387563853 hasRelatedWork W2153861281 @default.
• W4387563853 hasRelatedWork W2567243979 @default.
• W4387563853 hasRelatedWork W2763011410 @default.
• W4387563853 hasRelatedWork W2975239558 @default.
• W4387563853 hasRelatedWork W3205203927 @default.
• W4387563853 hasRelatedWork W4211078426 @default.
• W4387563853 hasRelatedWork W4214479279 @default.
• W4387563853 hasRelatedWork W4311331719 @default.
• W4387563853 hasRelatedWork W4387302241 @default.
• W4387563853 isParatext "false" @default.
• W4387563853 isRetracted "false" @default.
• W4387563853 workType "article" @default.