Susan J. Ward
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View article: Predicting trajectories of the north star ambulatory assessment total score in Duchenne muscular dystrophy
Predicting trajectories of the north star ambulatory assessment total score in Duchenne muscular dystrophy Open
The North Star Ambulatory Assessment (NSAA) is a widely used functional endpoint in drug development for ambulatory patients with Duchenne muscular dystrophy (DMD). Accurately predicting NSAA total score trajectories is important for desig…
View article: Meaningful changes in motor function in Duchenne muscular dystrophy (DMD): A multi-center study
Meaningful changes in motor function in Duchenne muscular dystrophy (DMD): A multi-center study Open
Evaluations of treatment efficacy in Duchenne muscular dystrophy (DMD), a rare genetic disease that results in progressive muscle wasting, require an understanding of the ‘meaningfulness’ of changes in functional measures. We estimated the…
View article: Functional trajectories before and after loss of ambulation in Duchenne muscular dystrophy and implications for clinical trials
Functional trajectories before and after loss of ambulation in Duchenne muscular dystrophy and implications for clinical trials Open
This study examined functional trajectories of subjects during the transition phase between ambulatory and non-ambulatory Duchenne muscular dystrophy (DMD) to inform clinical trial designs for new therapeutics. Ambulatory, pulmonary, and u…
View article: 269th ENMC international workshop: 10 years of clinical trials in Duchenne muscular dystrophy – What have we learned? 9–11 December 2022, Hoofddorp, The Netherlands
269th ENMC international workshop: 10 years of clinical trials in Duchenne muscular dystrophy – What have we learned? 9–11 December 2022, Hoofddorp, The Netherlands Open
There are multiple avenues for therapeutic development in Duchenne muscular dystrophy (DMD), which are highlighted in the first section of this report for the "10 years of Clinical trials in DMD - What have we learned?" workshop. This repo…
View article: (451) Human Rights and Sexual Health to Reproductive Health, A Research Project on Massive Open One line Courses (MOOCs)
(451) Human Rights and Sexual Health to Reproductive Health, A Research Project on Massive Open One line Courses (MOOCs) Open
Introduction Faced with alarming and persistent reproductive health indicators, the raise in refusal of oral contraception by young people and the decline in access to abortion in some developed countries, United states more precisely, UNE…
View article: <i>DMD</i> Genotypes and Motor Function in Duchenne Muscular Dystrophy
<i>DMD</i> Genotypes and Motor Function in Duchenne Muscular Dystrophy Open
These findings suggest the viability of trial designs incorporating genotypically mixed or unmatched controls for up to 12 months in duration for motor function outcomes, which would ease recruitment challenges and reduce numbers of patien…
View article: Real-world and natural history data for drug evaluation in Duchenne muscular dystrophy: suitability of the North Star Ambulatory Assessment for comparisons with external controls
Real-world and natural history data for drug evaluation in Duchenne muscular dystrophy: suitability of the North Star Ambulatory Assessment for comparisons with external controls Open
View article: Suitability of external controls for drug evaluation in Duchenne muscular dystrophy
Suitability of external controls for drug evaluation in Duchenne muscular dystrophy Open
OBJECTIVE: To evaluate the suitability of real-world data (RWD) and natural history data (NHD) for use as external controls in drug evaluations for ambulatory Duchenne muscular dystrophy (DMD). METHODS: The consistency of changes in the 6-…
View article: Intermediate models for Δ4SC time in each data source.
Intermediate models for Δ4SC time in each data source. Open
View article: Prognostic factors for changes in the timed 4-stair climb in patients with Duchenne muscular dystrophy, and implications for measuring drug efficacy: A multi-institutional collaboration
Prognostic factors for changes in the timed 4-stair climb in patients with Duchenne muscular dystrophy, and implications for measuring drug efficacy: A multi-institutional collaboration Open
The timed 4-stair climb (4SC) assessment has been used to measure function in Duchenne muscular dystrophy (DMD) practice and research. We sought to identify prognostic factors for changes in 4SC, assess their consistency across data source…
View article: Intermediate models for Δ4SC velocity in each data source.
Intermediate models for Δ4SC velocity in each data source. Open
View article: Categorising trajectories and individual item changes of the North Star Ambulatory Assessment in patients with Duchenne muscular dystrophy
Categorising trajectories and individual item changes of the North Star Ambulatory Assessment in patients with Duchenne muscular dystrophy Open
Functional variability among boys with Duchenne muscular dystrophy (DMD) is well recognised and complicates interpretation of clinical studies. We hypothesised that boys with DMD could be clustered into groups sharing similar trajectories …
View article: Pharmacokinetics and pharmacodynamics of sublingual sufentanil for postoperative pain management
Pharmacokinetics and pharmacodynamics of sublingual sufentanil for postoperative pain management Open
Summary Achieving successful treatment of postoperative pain remains a challenge. Recently, a sufentanil sublingual tablet system has been developed for treatment of moderate‐to‐severe postoperative pain. The phenylpiperidine sufentanil is…
View article: Corrigendum to “Categorizing natural history trajectories of ambulatory function measured by the 6-minute walk distance in patients with Duchenne muscular dystrophy” [Neuromuscular Disorders 26/9 (2016) 576–583]
Corrigendum to “Categorizing natural history trajectories of ambulatory function measured by the 6-minute walk distance in patients with Duchenne muscular dystrophy” [Neuromuscular Disorders 26/9 (2016) 576–583] Open
View article: Categorizing natural history trajectories of ambulatory function measured by the 6-minute walk distance in patients with Duchenne muscular dystrophy
Categorizing natural history trajectories of ambulatory function measured by the 6-minute walk distance in patients with Duchenne muscular dystrophy Open
View article: The clinical and operational consequences of prolonged occupancy strain and the use of non-intensive care beds to deliver critical care in a central london teaching hospital
The clinical and operational consequences of prolonged occupancy strain and the use of non-intensive care beds to deliver critical care in a central london teaching hospital Open
Since 2000, there has been a marked reduction in non-clinical critical care transfers in England. ICNARC have reported that this has been associated with a reduction in case-mix adjusted mortality (1). One consequence has been the need to …
View article: Virtual research visits and direct-to-consumer genetic testing in Parkinson’s disease
Virtual research visits and direct-to-consumer genetic testing in Parkinson’s disease Open
Objective The purpose of this study was to conduct a proof-of-concept study to evaluate remote recruitment and assessment of individuals (“virtual research visits”) with Parkinson's disease who have pursued direct-to-consumer genetic testi…