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In a Ph 3 study (DMD114044), a 10 m treatment difference (NS) on 6-min walking distance (6MWD) in favour of DRIS was seen in the overall study population but there was a treatment benefit of 21 m in a pre-specified subset of boys aged ⩽7y (n = 79), suggesting that younger, less progressed boys could benefit more from DRIS in terms of ambulatory endpoints vs older boys in whom DMD is more advanced.
These studies will shed important light on the role of DRIs in the treatment of HF.
Protein intake was found to be below the DRIs in all three groups, being far lower in the active CD group than in the inactive CD and control group (68.2 % vs. 17.2 % and 14.3 % below the DRIs, respectively).
Although calcium and magnesium intake was low, in comparison with the DRIs, in all three groups, none of the groups showed serum calcium deficiency or serum magnesium deficiency (Fig. 4).
Macronutrient intake was found to be below the DRIs in 9.1 % of the patients with active CD and in 5.7 % of the controls; no decrease in carbohydrate intake was observed in the patients with inactive CD.
We evaluated 150 healthy children (male 50%, mean age 10 years); at baseline a low VitD intake was observed in all subjects (median 0.79 μg/die, IQR 1.78; range 0.01-5.02); this condition was associated with Ca2+ intake <70% of the DRIs in 82 subjects (55%).
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In an open-label extension (OLE; DMD114349) of two 48-wk placebo (PBO -controlled trials (DMD114117, DMD114044), 96 wks' DRIS resulted in a clinically meaningful difference from 48 wks' PBO -controlleddy) followed by DRIS (OLE) in 6MWD of 46 m (n = 113; completrials wks', DMD114117).
For the first few months Chester and Dris shared a dingy rental house near Bowles in Asilah.
DRIS is a 2′-O-methyl-phosphorothioate antisense oligonucleotide designed to skip exon 51 in dystrophin pre-mRNA of boys with DMD.
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