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Search: L773:2042 6313 > (2020-2024) > Ataluren delays los...

  • McDonald, C. M. (author)

Ataluren delays loss of ambulation and respiratory decline in nonsense mutation Duchenne muscular dystrophy patients

  • Article/chapterEnglish2022

Publisher, publication year, extent ...

  • Becaris Publishing Limited,2022

Numbers

  • LIBRIS-ID:oai:gup.ub.gu.se/312205
  • https://gup.ub.gu.se/publication/312205URI
  • https://doi.org/10.2217/cer-2021-0196DOI
  • http://kipublications.ki.se/Default.aspx?queryparsed=id:148098385URI

Supplementary language notes

  • Language:English

Part of subdatabase

Classification

  • Subject category:ref swepub-contenttype
  • Subject category:art swepub-publicationtype

Notes

  • Aim: We investigated the effect of ataluren plus standard of care (SoC) on age at loss of ambulation (LoA) and respiratory decline in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD) versus patients with DMD on SoC alone. Patients & methods: Study 019 was a long-term Phase III study of ataluren safety in nmDMD patients with a history of ataluren exposure. Propensity score matching identified Study 019 and CINRG DNHS patients similar in disease progression predictors. Results & conclusion: Ataluren plus SoC was associated with a 2.2-year delay in age at LoA (p = 0.0006), and a 3.0-year delay in decline of predicted forced vital capacity to <60% in nonambulatory patients (p = 0.0004), versus SoC. Ataluren plus SoC delays disease progression and benefits ambulatory and nonambulatory patients with nmDMD. ClinicalTrials.gov: .

Subject headings and genre

Added entries (persons, corporate bodies, meetings, titles ...)

  • Muntoni, F. (author)
  • Penematsa, V. (author)
  • Jiang, J. (author)
  • Kristensen, A. (author)
  • Bibbiani, F. (author)
  • Goodwin, E. (author)
  • Gordish-Dressman, H. (author)
  • Morgenroth, L. (author)
  • Werner, C. (author)
  • Li, J. (author)
  • Able, R. (author)
  • Trifillis, P. (author)
  • Tulinius, Mar,1953Gothenburg University,Göteborgs universitet,Institutionen för kliniska vetenskaper, Avdelningen för pediatrik,Institute of Clinical Sciences, Department of Pediatrics(Swepub:gu)xtulim (author)
  • Göteborgs universitetInstitutionen för kliniska vetenskaper, Avdelningen för pediatrik (creator_code:org_t)

Related titles

  • In:Journal of Comparative Effectiveness Research: Becaris Publishing Limited11:32042-63052042-6313

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