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Natural history of disease

Determining minimal clinically important differences in the North Star Ambulatory Assessment (NSAA) for patients with Duchenne muscular dystrophy

Vandana Ayyar Gupta, Jacqueline M. Pitchforth, [ ... ], on behalf of the iMDEX Consortium and the U.K. NorthStar Clinical Network

Is information provided within chronic subdural haematoma education resources adequate? A scoping review

Conor S. Gillespie, Samuel Khanna, [ ... ], Benjamin M. Davies

Revised Hammersmith Scale for spinal muscular atrophy: Inter and intra-rater reliability and agreement

Danielle Ramsey, Gita Ramdharry, [ ... ], on behalf of the SMA REACH UK network

Novel approaches to analysis of the North Star Ambulatory Assessment (NSAA) in Duchenne muscular dystrophy (DMD): Observations from a phase 2 trial

Francesco Muntoni, Michela Guglieri, [ ... ], Eugenio Mercuri

Predictive models in SMA II natural history trajectories using machine learning: A proof of concept study

Giorgia Coratti, Jacopo Lenkowicz, [ ... ], Eugenio Mercuri

Bayesian modeling and simulation to inform rare disease drug development early decision-making: Application to Duchenne muscular dystrophy

Janelle L. Lennie, John T. Mondick, Marc R. Gastonguay

The impact of disease-related knowledge on perceptions of stigma among patients with Hepatitis C Virus (HCV) infection

M. Elle Saine, Julia E. Szymczak, [ ... ], Vincent Lo Re III

North Star Ambulatory Assessment changes in ambulant Duchenne boys amenable to skip exons 44, 45, 51, and 53: A 3 year follow up

Giorgia Coratti, Marika Pane, [ ... ], on behalf on the International DMD Group and the iMDEX Consortium

Estimating the disease burden of Korean type 2 diabetes mellitus patients considering its complications

Juyoung Kim, Seok-Jun Yoon, Min-Woo Jo

Change in global transmission rates of COVID-19 through May 6 2020

Ethan Obie Romero-Severson, Nick Hengartner, Grant Meadors, Ruian Ke

A model-based framework for chronic hepatitis C prevalence estimation

Abdullah Hamadeh, Zeny Feng, Murray Krahn, William W. L. Wong

Categorising trajectories and individual item changes of the North Star Ambulatory Assessment in patients with Duchenne muscular dystrophy

Francesco Muntoni, Joana Domingos, [ ... ], Susan J. Ward

Long-term natural history data in Duchenne muscular dystrophy ambulant patients with mutations amenable to skip exons 44, 45, 51 and 53

Claudia Brogna, Giorgia Coratti, [ ... ], on behalf on the International DMD group

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