Details:

Alternative Title:
Neuromuscul Disord
Publisher's site:
http://dx.doi.org/10.1016/j.nmd.2017.06.002
Personal Author:
Gissy, Jacob J.; Johnson, Teresa; Fox, Deborah J.; Kumar, Anil; Ciafaloni, Emma; ... More +
Gissy, Jacob J.; Johnson, Teresa; Fox, Deborah J.; Kumar, Anil; Ciafaloni, Emma; van Essen, Anthonie J.; Peay, Holly L.; Martin, Ann; Lucas, Ann; Finkel, Richard S.; Less -
Corporate Authors:
MD STARnet;
Description:
Individuals with Duchenne muscular dystrophy (DMD) often exhibit delayed motor and cognitive development, including delayed onset of ambulation. Data on age when loss of independent ambulation occurs are well established for DMD; however, age at onset of walking has not been well described. We hypothesize that an effective medication given in early infancy would advance the age when walking is achieved so that it is closer to age-matched norms, and that this discrete event could serve as the primary outcome measure in a clinical trial. This study examined three data sets, Muscular Dystrophy Surveillance, Tracking, and Research Network (MD STARnet); Dutch Natural History Survey (DNHS); and Parent Project Muscular Dystrophy (PPMD). The distribution of onset of ambulation in DMD (mean ± SD) and median age, in months, at the onset of ambulation was 17.3 (±5.5) and 16.0 in MD STARnet, 21.8 (±7.1) and 20.0 in DNHS, and 16.1 (±4.4) and 15 in PPMD. Age of ambulation in these data sets were all significantly later (P <0.001) than the corresponding age for typically developing boys, 12.1 (±1.8). A hypothetical clinical trial study design and power analyses are presented based on these data.
Subject:
[+]
Age Of Onset
Ambulation
Article
Child
Clinical Trial
Clinical Trials As Topic
DMD
Duchenne
Endpoint Determination
Humans
Male
Motor Development
Muscular Dystrophy, Duchenne
Walking

Source:
Neuromuscul Disord. 27(10):905-910.;
Pubmed ID:
28739181
Pubmed Central ID:
PMC5628390
Document Type:
Journal Article;
Funding:
U01 DD000392/DD/NCBDD CDC HHS/United States; U01 DD000187/DD/NCBDD CDC HHS/United States; U01 DD001123/DD/NCBDD CDC HHS/United States; U01 DD000189/DD/NCBDD CDC HHS/United States; CC999999/Intramural CDC HHS/United States; ... More +
U01 DD000392/DD/NCBDD CDC HHS/United States; U01 DD000187/DD/NCBDD CDC HHS/United States; U01 DD001123/DD/NCBDD CDC HHS/United States; U01 DD000189/DD/NCBDD CDC HHS/United States; CC999999/Intramural CDC HHS/United States; U01 DD000191/DD/NCBDD CDC HHS/United States; U01 DD001119/DD/NCBDD CDC HHS/United States; U01 DD000190/DD/NCBDD CDC HHS/United States; Less -
Collection(s):
CDC Public Access
Main Document Checksum:
[+]
urn:sha256:68e6c7f309e5a8cf50f94973f4c782cd0dd24e203840e32d5519e4bdef196a99
File Type:

Supporting Files

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