Matches in SemOpenAlex for { <https://semopenalex.org/work/W4384208076> ?p ?o ?g. }
Showing items 1 to 92 of
92
with 100 items per page.
- W4384208076 abstract "Spinal muscular atrophy (SMA) is a severe hereditary lower motor neuron disorder characterised by degeneration of alpha motor neurons in the spinal cord, resulting in progressive weakness and paralysis of proximal muscles. A systematic literature search was carried out by using PRISMA guidelines and searching through different databases that could provide findings of evidence on the health outcomes of the approved therapies for the management of paediatric SMA type 1 regarding efficacy with follow-up in terms of motor and respiratory functions and the tolerability and incidence of adverse drug reactions (ADRs) post-treatment from real-world publications. Half of the publications (50%) had a prospective observational design. Eight studies (66.7%) assessed nusinersen, and three studies (25%) assessed onasemnogene abeparvovec with a duration of follow-up ranging from 6 months to 3 years to evaluate the motor and respiratory functions using different assessment tools, hospitalisation rates, and the tolerability and incidence of ADRs post-treatment. The three currently approved treatments for SMA type 1 provided good support and health outcomes in terms of motor function, respiratory outcomes, reduction of hospitalisations, and improvement of survival. Nevertheless, uncertainties regarding continued improvement after long-term illness and the generalizability of results are still unknown." @default.
- W4384208076 created "2023-07-14" @default.
- W4384208076 creator A5033520648 @default.
- W4384208076 creator A5043885224 @default.
- W4384208076 date "2023-07-01" @default.
- W4384208076 modified "2023-09-27" @default.
- W4384208076 title "Evaluation of the Therapeutic Efficacy and Tolerability of Current Drug Treatments on the Clinical Outcomes of Paediatric Spinal Muscular Atrophy Type 1: A Systematic Review" @default.
- W4384208076 cites W1980537418 @default.
- W4384208076 cites W2007954199 @default.
- W4384208076 cites W2036089303 @default.
- W4384208076 cites W2143197369 @default.
- W4384208076 cites W2168402647 @default.
- W4384208076 cites W2558189204 @default.
- W4384208076 cites W2769295220 @default.
- W4384208076 cites W2895752535 @default.
- W4384208076 cites W2904099718 @default.
- W4384208076 cites W2920823484 @default.
- W4384208076 cites W3005454283 @default.
- W4384208076 cites W3014541336 @default.
- W4384208076 cites W3030807865 @default.
- W4384208076 cites W3113507839 @default.
- W4384208076 cites W3119526862 @default.
- W4384208076 cites W3123749546 @default.
- W4384208076 cites W3135935053 @default.
- W4384208076 cites W3159668658 @default.
- W4384208076 cites W3160334825 @default.
- W4384208076 cites W3177991868 @default.
- W4384208076 cites W3186720955 @default.
- W4384208076 cites W4200169031 @default.
- W4384208076 cites W4214728032 @default.
- W4384208076 cites W4220956535 @default.
- W4384208076 cites W4225124381 @default.
- W4384208076 cites W4306150167 @default.
- W4384208076 doi "https://doi.org/10.1016/j.prrv.2023.06.004" @default.
- W4384208076 hasPubMedId "https://pubmed.ncbi.nlm.nih.gov/37563072" @default.
- W4384208076 hasPublicationYear "2023" @default.
- W4384208076 type Work @default.
- W4384208076 citedByCount "0" @default.
- W4384208076 crossrefType "journal-article" @default.
- W4384208076 hasAuthorship W4384208076A5033520648 @default.
- W4384208076 hasAuthorship W4384208076A5043885224 @default.
- W4384208076 hasConcept C114614502 @default.
- W4384208076 hasConcept C126322002 @default.
- W4384208076 hasConcept C141071460 @default.
- W4384208076 hasConcept C161921814 @default.
- W4384208076 hasConcept C177713679 @default.
- W4384208076 hasConcept C1862650 @default.
- W4384208076 hasConcept C187212893 @default.
- W4384208076 hasConcept C197934379 @default.
- W4384208076 hasConcept C23131810 @default.
- W4384208076 hasConcept C2778375690 @default.
- W4384208076 hasConcept C2778558090 @default.
- W4384208076 hasConcept C2779134260 @default.
- W4384208076 hasConcept C2780247198 @default.
- W4384208076 hasConcept C33923547 @default.
- W4384208076 hasConcept C535046627 @default.
- W4384208076 hasConcept C71924100 @default.
- W4384208076 hasConcept C99508421 @default.
- W4384208076 hasConceptScore W4384208076C114614502 @default.
- W4384208076 hasConceptScore W4384208076C126322002 @default.
- W4384208076 hasConceptScore W4384208076C141071460 @default.
- W4384208076 hasConceptScore W4384208076C161921814 @default.
- W4384208076 hasConceptScore W4384208076C177713679 @default.
- W4384208076 hasConceptScore W4384208076C1862650 @default.
- W4384208076 hasConceptScore W4384208076C187212893 @default.
- W4384208076 hasConceptScore W4384208076C197934379 @default.
- W4384208076 hasConceptScore W4384208076C23131810 @default.
- W4384208076 hasConceptScore W4384208076C2778375690 @default.
- W4384208076 hasConceptScore W4384208076C2778558090 @default.
- W4384208076 hasConceptScore W4384208076C2779134260 @default.
- W4384208076 hasConceptScore W4384208076C2780247198 @default.
- W4384208076 hasConceptScore W4384208076C33923547 @default.
- W4384208076 hasConceptScore W4384208076C535046627 @default.
- W4384208076 hasConceptScore W4384208076C71924100 @default.
- W4384208076 hasConceptScore W4384208076C99508421 @default.
- W4384208076 hasLocation W43842080761 @default.
- W4384208076 hasLocation W43842080762 @default.
- W4384208076 hasOpenAccess W4384208076 @default.
- W4384208076 hasPrimaryLocation W43842080761 @default.
- W4384208076 hasRelatedWork W1515376677 @default.
- W4384208076 hasRelatedWork W2013301809 @default.
- W4384208076 hasRelatedWork W2121965889 @default.
- W4384208076 hasRelatedWork W2300951885 @default.
- W4384208076 hasRelatedWork W2803597081 @default.
- W4384208076 hasRelatedWork W2914730463 @default.
- W4384208076 hasRelatedWork W2996406693 @default.
- W4384208076 hasRelatedWork W3118156846 @default.
- W4384208076 hasRelatedWork W4235322919 @default.
- W4384208076 hasRelatedWork W1278871635 @default.
- W4384208076 isParatext "false" @default.
- W4384208076 isRetracted "false" @default.
- W4384208076 workType "article" @default.