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Bettina M. Cockroft

Observed names: Bettina Cockroft, Bettina M Cockroft, Cockroft, Bettina M.
OpenAlex ID: A5038664397
A Phase 2, Double-Blind, Randomized, Dose-Ranging Trial Of Reldesemtiv In Patients With ALS
2020 · Jeremy M. Shefner, Jinsy A. Andrews, et al. · Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration · 57
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A phase III trial of tirasemtiv as a potential treatment for amyotrophic lateral sclerosis
2019 · Jeremy M. Shefner, Merit E. Cudkowicz, et al. · Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration · 47
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Reldesemtiv in Patients with Spinal Muscular Atrophy: a Phase 2 Hypothesis-Generating Study
2021 · Stacy A. Rudnicki, Jinsy A. Andrews, et al. · Neurotherapeutics · 46
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Giroctocogene fitelparvovec gene therapy for severe hemophilia A: 104-week analysis of the phase 1/2 Alta study
2023 · Andrew D. Leavitt, Barbara A. Konkle, et al. · Blood · 43
Updated Results of a Phase 1/2 Clinical Study of Zinc Finger Nuclease-Mediated Editing of BCL11A in Autologous Hematopoietic Stem Cells for Transfusion-Dependent Beta Thalassemia
2021 · Mark C. Walters, Angela R. Smith, et al. · Blood · 24
Updated Follow-up of the Alta Study, a Phase 1/2 Study of Giroctocogene Fitelparvovec (SB-525) Gene Therapy in Adults with Severe Hemophilia a
2020 · Andrew D. Leavitt, Barbara A. Konkle, et al. · Blood · 22
Zinc finger nuclease-mediated gene editing in hematopoietic stem cells results in reactivation of fetal hemoglobin in sickle cell disease
2024 · Samuel Lessard, Pauline Rimmelé, et al. · Scientific Reports · 21
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Updated Results of the Alta Study, a Phase 1/2 Study of Giroctocogene Fitelparvovec (PF-07055480/SB-525) Gene Therapy in Adults with Severe Hemophilia a
2021 · Nathan Visweshwar, Thomas J. Harrington, et al. · Blood · 19
Interim Safety and Efficacy Results from a Phase 1/2 Study of Zinc Finger Nuclease-Modified Autologous Hematopoietic Stem Cells for Sickle Cell Disease (PRECIZN-1)
2022 · Asif Alavi, Mehrdad Abedi, et al. · Blood · 15
COURAGE-ALS: a randomized, double-blind phase 3 study designed to improve participant experience and increase the probability of success
2023 · Jeremy M. Shefner, Ammar Al-Chalabi, et al. · Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration · 14
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Updated Results of the Alta Study, a Phase 1/2 Study of Giroctocogene Fitelparvovec (PF-07055480/SB-525) Gene Therapy in Adults with Severe Hemophilia Α
2022 · Adam Giermasz, Nathan Visweshwar, et al. · Blood · 7
Four-Year Follow-up of the Alta Study, a Phase 1/2 Study of Giroctocogene Fitelparvovec (PF-07055480/SB-525) Gene Therapy in Adults with Severe Hemophilia Α
2023 · Thomas J. Harrington, Adam Giermasz, et al. · Blood · 5
Noninvasive ventilation use by patients enrolled in VITALITY-ALS
2021 · Stacy A. Rudnicki, Jinsy A. Andrews, et al. · Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration · 5
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Prescription and acceptance of durable medical equipment in FORTITUDE-ALS, a study of reldesemtiv in ALS: post hoc analyses of a randomized, double-blind, placebo-controlled clinical trial
2021 · Stacy A. Rudnicki, Jinsy A. Andrews, et al. · Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration · 3
Correction to: Reldesemtiv in Patients with Spinal Muscular Atrophy: a Phase 2 Hypothesis-Generating Study
2021 · Stacy A. Rudnicki, Jinsy A. Andrews, et al. · Neurotherapeutics · 3
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A phase I/II multicenter gene therapy clinical study for Fabry disease
2021 · Jaya Ganesh, Ozlem Goker-Alpan, et al. · Molecular Genetics and Metabolism · 3
STAAR, a phase I/II study of isaralgagene civaparvovec (ST-920) gene therapy in adults with Fabry disease: Dose escalation phase results
2023 · Robert J. Hopkin, Jaya Ganesh, et al. · Molecular Genetics and Metabolism · 2
Correlation between Slow Vital Capacity Measured in the Home and in the Clinic for Patients with Amyotrophic Lateral Sclerosis (P4.4-002)
2019 · Lisa Meng, Stacy Rudnicki, et al. · Neurology · 2
Update of FORTITUDE-ALS: A Phase 2, Double-blind, Randomized, Placebo-Controlled, Study to Evaluate Efficacy, Safety and Tolerability of Reldesemtiv in Patients with Amyotrophic Lateral Sclerosis (S5.002)
2019 · Jeremy Shefner, Jinsy Andrews, et al. · Neurology · 1
Gene therapy with giroctocogene fitelparvovec for people with severe hemophilia A: a plain language summary of the 2-year results of a 5-year study
2025 · Andrew D. Leavitt, Barbara A. Konkle, et al. · Future Rare Diseases
Works count: 22
Citation count: 338
H-index: 10
I10-index: 10
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