Suchergebnisse - "McDonald, Craig M"

PubPharm (66)

1

Survival among patients receiving eteplirsen for up to 8 years for the treatment of Duchenne muscular dystrophy and contextualization with natural history controls

enthalten in: Muscle & nerve | 2024

von Iff, J. | Done, N. | Tuttle, E. | +6

2

Practical Considerations for Delandistrogene Moxeparvovec Gene Therapy in Patients With Duchenne Muscular Dystrophy

enthalten in: Pediatric neurology | 2024

von Mendell, J. | Proud, C. | Zaidman, C. | +8

3

Gait Event Detection and Travel Distance Using Waist-Worn Accelerometers across a Range of Speeds : Automated Approach

enthalten in: Sensors (Basel, Switzerland) | 2024

von Ramli, A. | Liu, X. | Berndt, K. | +11

4

The IAAM LTBP4 Haplotype is Protective Against Dystrophin-Deficient Cardiomyopathy

enthalten in: Journal of neuromuscular diseases | 2024

von Bello, L. | Sabbatini, D. | Fusto, A. | +36

5

Disease progression rates in ambulatory Duchenne muscular dystrophy by steroid type, patient age and functional status

enthalten in: Journal of comparative effectiveness research | 2023

von McDonald, C. | Marden, J. | Shieh, P. | +8

6

Efficacy and Safety of Viltolarsen in Boys With Duchenne Muscular Dystrophy : Results From the Phase 2, Open-Label, 4-Year Extension Study

enthalten in: Journal of neuromuscular diseases | 2023

von Clemens, P. | Rao, V. | Connolly, A. | +8

7

Assessing the value of delandistrogene moxeparvovec (SRP-9001) gene therapy in patients with Duchenne muscular dystrophy in the United States

enthalten in: Journal of market access & health policy | 2023

von Klimchak, A. | Sedita, L. | Rodino-Klapac, L. | +4

8

Functional and Clinical Outcomes Associated with Steroid Treatment among Non-ambulatory Patients with Duchenne Muscular Dystrophy1

enthalten in: Journal of neuromuscular diseases | 2023

von McDonald, C. | Mayer, O. | Hor, K. | +12

9

Modeling Early Heterogeneous Rates of Progression in Boys with Duchenne Muscular Dystrophy

enthalten in: Journal of neuromuscular diseases | 2023

von Fang, Y. | McDonald, C. | Clemens, P. | +5

10

Expression of SRP-9001 dystrophin and stabilization of motor function up to 2 years post-treatment with delandistrogene moxeparvovec gene therapy in individuals with Duchenne muscular dystrophy

enthalten in: Frontiers in cell and developmental biology | 2023

von Mendell, J. | Shieh, P. | McDonald, C. | +17

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