Suchergebnisse - "Finkel, Richard"

PubPharm (377)

1

Determining minimal clinically important differences in the Hammersmith Functional Motor Scale Expanded for untreated spinal muscular atrophy patients : An international study

enthalten in: European journal of neurology | 2024

von Coratti, G. | Bovis, F. | Pera, M. | +44

2

2-year Change in Revised Hammersmith Scale scores in a large cohort of untreated paediatric type 2 and 3 SMA participants

enthalten in: bioRxiv.org | 2024

von Stimpson, G. | Ramsey, D. | Wolfe, A. | +29

3

Bulbar function in spinal muscular atrophy (SMA) : State of art and new challenges. 21st July 2023, Rome, Italy

enthalten in: Neuromuscular disorders : NMD | 2024

von McGrattan, K. | Cerchiari, A. | Conway, E. | +41

4

Disease Trajectories in the Revised Hammersmith Scale in a Cohort of Untreated Patients with Spinal Muscular Atrophy types 2 and 3

enthalten in: Journal of neuromuscular diseases | 2024

von Wolfe, A. | Stimpson, G. | Ramsey, D. | +32

5

Efficacy and Safety of Vamorolone Over 48 Weeks in Boys With Duchenne Muscular Dystrophy : A Randomized Controlled Trial

enthalten in: Neurology | 2024

von Dang, U. | Damsker, J. | Guglieri, M. | +41

6

Can the CHOP-INTEND be used as An Outcome Measure in the First Months of Age? Implications for Clinical Trials and Real World Data

enthalten in: Journal of neuromuscular diseases | 2024

von Cutrona, C. | de Sanctis, R. | Coratti, G. | +14

7

The Clinical Development of Taldefgrobep Alfa : An Anti-Myostatin Adnectin for the Treatment of Duchenne Muscular Dystrophy

enthalten in: Neurology and therapy | 2024

von Muntoni, F. | Byrne, B. | McMillan, H. | +114

8

Real-World Outcomes in Patients with Spinal Muscular Atrophy Treated with Onasemnogene Abeparvovec Monotherapy : Findings from the RESTORE Registry

enthalten in: Journal of neuromuscular diseases | 2024

von Servais, L. | Day, J. | De Vivo, D. | +16

9

Draft Guidance for Industry Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, and Related Dystrophinopathies - Developing Potential Treatments for the Entire Spectrum of Disease

enthalten in: Journal of neuromuscular diseases | 2024

von McDonald, C. | Camino, E. | Escandon, R. | +8

10

Combination disease-modifying treatment in spinal muscular atrophy : A proposed classification

enthalten in: Annals of clinical and translational neurology | 2023

von Proud, C. | Mercuri, E. | Finkel, R. | +11

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