| Efficacy and safety of onasemnogene abeparvovec in children with spinal muscular atrophy type 1: real-world evidence from 6 infusion centres in the United Kingdom V Gowda, M Atherton, A Murugan, L Servais, J Sheehan, E Standing, ... The Lancet Regional Health–Europe 37, 2024 | 12 | 2024 |
| Determining minimal clinically important differences in the North Star Ambulatory Assessment (NSAA) for patients with Duchenne muscular dystrophy V Ayyar Gupta, JM Pitchforth, J Domingos, D Ridout, M Iodice, C Rye, ... Plos one 18 (4), e0283669, 2023 | 11 | 2023 |
| Safety and efficacy of tamoxifen in boys with Duchenne muscular dystrophy (TAMDMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial BC Henzi, S Schmidt, S Nagy, D Rubino-Nacht, S Schaedelin, ... The Lancet Neurology 22 (10), 890-899, 2023 | 9 | 2023 |
| Longitudinal changes in respiratory and upper limb function in a pediatric type III spinal muscular atrophy cohort after loss of ambulation A Wolfe, M Scoto, E Milev, R Muni Lofra, L Abbott, R Wake, A Rohwer, ... Muscle & Nerve 64 (5), 545-551, 2021 | 8 | 2021 |
| 2-year change in revised Hammersmith scale scores in a large cohort of untreated paediatric type 2 and 3 SMA participants G Stimpson, D Ramsey, A Wolfe, A Mayhew, M Scoto, G Baranello, ... Journal of Clinical Medicine 12 (5), 1920, 2023 | 6 | 2023 |
| Quantifying variability in motor function in Duchenne muscular dystrophy: UK centiles for the NorthStar ambulatory assessment, 10 m walk run velocity and rise from floor … G Stimpson, D Ridout, A Wolfe, E Milev, E O’Reilly, A Manzur, A Sarkozy, ... Journal of Neuromuscular Diseases, 1-14, 2024 | 4 | 2024 |
| Disease Trajectories in the revised hammersmith scale in a cohort of untreated patients with spinal muscular atrophy types 2 and 3 A Wolfe, G Stimpson, D Ramsey, G Coratti, S Dunaway Young, A Mayhew, ... Journal of Neuromuscular Diseases 11 (3), 665-677, 2024 | 1 | 2024 |
| Assessment of the upper limb function, strength, and mobility in treatment‐naive children with spinal muscular atrophy Types 2 and 3 E Milev, V Selby, A Wolfe, A Rohwer, R Tillmann, D Ramsey, M Iodice, ... Muscle & Nerve 69 (3), 340-348, 2024 | 1 | 2024 |
| P139 Correlation and validation of the North Star Ambulatory Assessment, timed test and motor function measure centiles for boys with Duchenne muscular dystrophy E Milev, G Stimpson, M van der Holst, A Wolfe, E O'Reilly, A Manzur, ... Neuromuscular Disorders 33, S82, 2023 | 1 | 2023 |
| 74 Hypermobility in young boys with duchenne muscular dystrophy and the effect on attainment of walking age and North star ambulatory assessment functional skills L Abbott, V Selby, VA Guptar, S Wadsworth, A Wolfe, M Chesshyre, ... Archives of Disease in Childhood 105 (Suppl 2), A25-A26, 2020 | 1 | 2020 |
| Investigating temporal changes in percent predicted FVC and RULM score in non-ambulant SMA type III children A Wolfe, M Scoto, E Milev, R Lofra, A Rohwer, R Wake, A Mayhew, ... NEUROMUSCULAR DISORDERS 30, S102-S102, 2020 | 1 | 2020 |
| Understanding North Star Ambulatory Assessment total scores and their implications for standards of care using observational data G Stimpson, MK James, M Guglieri, A Wolfe, A Manzur, A Sarkozy, ... European Journal of Paediatric Neurology, 2024 | | 2024 |
| Spinal presentations in children with Spinal Muscular Atrophy type 1 following Gene Therapy treatment with onasemnogene abeparvovec–the SMA REACH UK network experience A Wolfe, J Sheehan, A Schofield, H Cranney, E O'Reilly, G Stimpson, ... Neuromuscular Disorders, 104451, 2024 | | 2024 |
| P219 Longitudinal disease progression in the Revised Hammersmith Scale in a cohort of untreated SMA 2 and 3 patients G Stimpson, A Wolfe, D Ramsey, E O'Reilly, A Rowher, RM Lofra, ... Neuromuscular Disorders 33, S90, 2023 | | 2023 |
| P75 Revised Hammersmith Scale item achievement by functional status in an international cohort of untreated SMA 2 and 3 patients D Ramsey, G Stimpson, A Wolfe, E O'Reilly, A Rowher, RM Lofra, ... Neuromuscular Disorders 33, S135, 2023 | | 2023 |
| P143 Centiles by age for the North Star ambulatory assessment and the associated timed items in glucocorticoid treated boys with Duchenne muscular dystrophy G Stimpson, D Ridout, A Wolfe, E Milev, E O'Reilly, A Manzur, T Cole, ... Neuromuscular Disorders 33, S83, 2023 | | 2023 |
| P136 Using the North Star and timed function test centiles in boys with Duchenne muscular dystrophy–a prospective study A Wolfe, G Stimpson, E Milev, E O'Reilly, A Manzur, A Sarkozy, F Muntoni, ... Neuromuscular Disorders 33, S81, 2023 | | 2023 |
| P214 Spinal presentations in children with spinal muscular atrophy type 1 following gene therapy treatment in the SMA-REACH UK network A Wolfe, J Sheehan, H Jungbluth Neuromuscular Disorders 33, S88, 2023 | | 2023 |
| P242 Understanding North Star Ambulatory Assessment total scores and their implications for standards of care G Stimpson, M James, M Guglieri, A Wolfe, A Manzur, G Baranello, ... Neuromuscular Disorders 33, S175, 2023 | | 2023 |
| P. 117 Collection of real-world evidence of nusinersen treatment for SMA patients through a national registry: description of the paediatric cohort in the UK A Rohwer, M Main, S Wadsworth, A Wolfe, M Madden, E Cavalcante, ... Neuromuscular Disorders 32, S90, 2022 | | 2022 |
