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Background: Duchenne muscular dystrophy (DMD) is caused by DMD gene mutations. Delandistrogene moxeparvovec is an investigational gene transfer therapy, developed to address the underlying cause of DMD. We report findings from Part 1 (52 weeks) of the two-part EMBARK trial (NCT05096221). Methods: Key inclusion criteria: Ambulatory patients aged ≥4-<8 years with a confirmed DMD mutation within exons 18–79 (inclusive); North Star Ambulatory Assessment (NSAA) score >16 and <29 at screening. Eligible patients were randomized 1:1 to intravenous delandistrogene moxeparvovec (1.33×1014 vg/kg) or placebo. The primary endpoint was change from baseline in NSAA total score to Week 52. Results: At Week 52 (n=125), the primary endpoint did not reach statistical significance, although there was a nominal difference in change from baseline in NSAA total score in the delandistrogene moxeparvovec (2.6, n=63) versus placebo groups (1.9, n=61). Key secondary endpoints (time to rise, micro-dystrophin expression, 10-meter walk/run) demonstrated treatment benefit in both age groups (4-5 and 6-7 years; p<0.05).There were no new safety signals, reinforcing the favorable and manageable safety profile observed to date. Conclusions: Based on the totality of functional assessments including the timed function tests, treatment with delandistrogene moxeparvovec indicates beneficial modification of disease trajectory.
The discovery of the first electromagnetic counterpart to a gravitational wave signal has generated follow-up observations by over 50 facilities world-wide, ushering in the new era of multi-messenger astronomy. In this paper, we present follow-up observations of the gravitational wave event GW170817 and its electromagnetic counterpart SSS17a/DLT17ck (IAU label AT2017gfo) by 14 Australian telescopes and partner observatories as part of Australian-based and Australian-led research programs. We report early- to late-time multi-wavelength observations, including optical imaging and spectroscopy, mid-infrared imaging, radio imaging, and searches for fast radio bursts. Our optical spectra reveal that the transient source emission cooled from approximately 6 400 K to 2 100 K over a 7-d period and produced no significant optical emission lines. The spectral profiles, cooling rate, and photometric light curves are consistent with the expected outburst and subsequent processes of a binary neutron star merger. Star formation in the host galaxy probably ceased at least a Gyr ago, although there is evidence for a galaxy merger. Binary pulsars with short (100 Myr) decay times are therefore unlikely progenitors, but pulsars like PSR B1534+12 with its 2.7 Gyr coalescence time could produce such a merger. The displacement (~2.2 kpc) of the binary star system from the centre of the main galaxy is not unusual for stars in the host galaxy or stars originating in the merging galaxy, and therefore any constraints on the kick velocity imparted to the progenitor are poor.
The serum antibody responses and 50% protective levels (PL50) of antibody were determined, using the SRH test, at one and twelve months post-vaccination in a group of student volunteers immunized with one of three dosages of a trivalent surface-antigen influenza virus vaccine, or with placebo.
It was found that, for the H3, H1 and B haemagglutinin components present in the vaccine, a dose of 6 μg HA elicited high serum antibody responses at one month post-immunization. High mean antibody levels and a high incidence of volunteers with PL50 values of antibody against each of the HA components of the vaccine remained in the volunteer group twelve months later. The results are discussed in relation to the vaccine dosage used and the nature of the population immunized.
The Romano-British settlement at Grandford lies northwest of the town of March, in the heart of the Fens of eastern England. It straddles the ‘Fen Causeway’, a Roman road that ran west—east across the Fens, and which probably originated at the legionary vexillation fortress at Longthorpe, near Peterborough, held between c. AD 48 and 61/62. Small-scale excavations between 1958 and 1968 demonstrated occupation for much of the Roman period, down to the later 4th century, beginning at least as early as c. AD 65 (Potter & Potter 1982). It was suggested on various grounds that the settlement niay have started life as a Roman fort, constructed in the aftermath of the great rebellion of AD 60-61, led by Boudicca, Queen of the Iceni (Potter 1981: 85-7).
George Dennis’ The cities and cemeteries of Etruria, a massive two-volume work of over 1000 pages, was published towards the end of 1848, the British Museum’s copy (now the British Library’s) being received on 18 January 1849. It was quickly acclaimed as a literary and archaeological masterpiece (Rhodes 1973: 52–5; Pallottino 1955: 126, n. 1), which brought the then little-known Etruscans to life in the most vivid of ways. The fruit, in Dennis’ word, of extensive travelling in Etruria between 1842 and 1847, and of much work in the libraries of, in particular, Rome, it remains 150 years later an indispensable topographical source. Indeed, a 2nd, revised, edition appeared in 1878 (reprinted in 1883, but misleadingly entitled a 3rd edition), and a further version of the 1848 volume was published in J.M. Dent’s highly regarded ‘Everyman’ series in 1907.
This paper reports excavations in 1977 and 1978 at the chapel and adjoining cemetery of St Patrick's, Heysham, and an architectural survey of the nearby church of St Peter's. The chapel initially comprised a single-celled, stone-built structure, plastered inside and out. Associated with it were pieces of painted plaster, two bearing letters, and, in all probability, a stone carved with a bird's head, perhaps of late seventh- to late eighth-century date. The chapel was subsequently enlarged. One burial included a bone comb of Anglo-Scandinavian type, and calibrated radiocarbon dates for three skeletons range between AD 960 and 1185.
This report was submitted for publication in March 1992.