Matches in SemOpenAlex for { <https://semopenalex.org/work/W2753993331> ?p ?o ?g. }
Showing items 1 to 56 of
56
with 100 items per page.
- W2753993331 endingPage "S216" @default.
- W2753993331 startingPage "S216" @default.
- W2753993331 abstract "Duchenne muscular dystrophy (DMD) is a progressive, degenerative neuromuscular disease caused by gene mutations that result in the absence or deficiency of dystrophin protein. Absence of dystrophin compromises the regenerative ability of muscle fibers, and, as muscle deterioration overtakes muscle growth, loss of upper limb function causes difficulties in performing daily activities and negatively impacts quality of life. Eteplirsen (Exondys 51; Sarepta Therapeutics, Cambridge, MA), the first FDA approved treatment for DMD in the US, is indicated for treatment of patients with DMD amenable to exon 51 skipping. Eteplirsen has not been approved for marketing outside of the United States. We evaluated long-term upper limb function outcomes in patients aged 7–13 years with DMD amenable to exon 51 skipping who participated in 2 eteplirsen clinical trials. Study 201, a 28-week trial, randomized patients to receive once-weekly intravenous eteplirsen infusions (30 mg/kg, n = 4; 50 mg/kg, n = 4) or placebo (n = 4) for 24 weeks. At week 25, all patients received open-label eteplirsen through week 28. In study 202, an open-label trial, all patients received once-weekly eteplirsen at the same dose as in study 201 through 240 weeks. Upper limb function tests included the 9-Hole Peg Test (seconds), a maximum voluntary isometric contraction test (MVICT) with a Quantitative Movement Assessment system to assess elbow flexion and extension and hand grip (kg). Study 201 enrolled 12 patients; all completed extension study 202. Following confirmation of dystrophin production, patients showed relative stability or minimal decline of upper limb function on all measures assessed through week 240. In conclusion, eteplirsen may slow the deterioration of upper arm function in patients with DMD amenable to exon 51 skipping." @default.
- W2753993331 created "2017-09-25" @default.
- W2753993331 creator A5002171875 @default.
- W2753993331 creator A5030393698 @default.
- W2753993331 creator A5034709372 @default.
- W2753993331 creator A5039453462 @default.
- W2753993331 creator A5041763238 @default.
- W2753993331 creator A5071844858 @default.
- W2753993331 date "2017-10-01" @default.
- W2753993331 modified "2023-10-05" @default.
- W2753993331 title "Effects of long-term eteplirsen treatment on upper limb function in patients with Duchenne muscular dystrophy: findings of two phase 2 clinical trials" @default.
- W2753993331 doi "https://doi.org/10.1016/j.nmd.2017.06.439" @default.
- W2753993331 hasPublicationYear "2017" @default.
- W2753993331 type Work @default.
- W2753993331 sameAs 2753993331 @default.
- W2753993331 citedByCount "1" @default.
- W2753993331 countsByYear W27539933312020 @default.
- W2753993331 crossrefType "journal-article" @default.
- W2753993331 hasAuthorship W2753993331A5002171875 @default.
- W2753993331 hasAuthorship W2753993331A5030393698 @default.
- W2753993331 hasAuthorship W2753993331A5034709372 @default.
- W2753993331 hasAuthorship W2753993331A5039453462 @default.
- W2753993331 hasAuthorship W2753993331A5041763238 @default.
- W2753993331 hasAuthorship W2753993331A5071844858 @default.
- W2753993331 hasConcept C126322002 @default.
- W2753993331 hasConcept C141071460 @default.
- W2753993331 hasConcept C2778943923 @default.
- W2753993331 hasConcept C2779030066 @default.
- W2753993331 hasConcept C535046627 @default.
- W2753993331 hasConcept C71924100 @default.
- W2753993331 hasConceptScore W2753993331C126322002 @default.
- W2753993331 hasConceptScore W2753993331C141071460 @default.
- W2753993331 hasConceptScore W2753993331C2778943923 @default.
- W2753993331 hasConceptScore W2753993331C2779030066 @default.
- W2753993331 hasConceptScore W2753993331C535046627 @default.
- W2753993331 hasConceptScore W2753993331C71924100 @default.
- W2753993331 hasLocation W27539933311 @default.
- W2753993331 hasOpenAccess W2753993331 @default.
- W2753993331 hasPrimaryLocation W27539933311 @default.
- W2753993331 hasRelatedWork W1978943632 @default.
- W2753993331 hasRelatedWork W2013779713 @default.
- W2753993331 hasRelatedWork W2028695000 @default.
- W2753993331 hasRelatedWork W2089569716 @default.
- W2753993331 hasRelatedWork W2110400822 @default.
- W2753993331 hasRelatedWork W2740435222 @default.
- W2753993331 hasRelatedWork W2754523463 @default.
- W2753993331 hasRelatedWork W2975608941 @default.
- W2753993331 hasRelatedWork W3159037196 @default.
- W2753993331 hasRelatedWork W4285603539 @default.
- W2753993331 hasVolume "27" @default.
- W2753993331 isParatext "false" @default.
- W2753993331 isRetracted "false" @default.
- W2753993331 magId "2753993331" @default.
- W2753993331 workType "article" @default.