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11 results on '"Darras, Basil T"'

1. Identifying Biomarkers of Spinal Muscular Atrophy for Further Development

Author

Glascock, Jacqueline, primary, Darras, Basil T., additional, Crawford, Thomas O., additional, Sumner, Charlotte J., additional, Kolb, Stephen J., additional, DiDonato, Christine, additional, Elsheikh, Bakri, additional, Howell, Kelly, additional, Farwell, Wildon, additional, Valente, Marta, additional, Petrillo, Marco, additional, Tingey, Jessica, additional, and Jarecki, Jill, additional

Published
2023
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2. Erratum to: Patients with Spinal Muscular Atrophy Type 1 Achieve and Maintain Bulbar Function Following Onasemnogene Abeparvovec Treatment

Author

McGrattan, Katlyn E., primary, Shell, Richard D., additional, Hurst-Davis, Rebecca, additional, Young, Sally Dunaway, additional, O’Brien, Eamonn, additional, Lavrov, Arseniy, additional, Wallach, Shiri, additional, LaMarca, Nicole, additional, Reyna, Sandra P., additional, and Darras, Basil T., additional

Published
2023
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3. Intrathecal Onasemnogene Abeparvovec for Sitting, Nonambulatory Patients with Spinal Muscular Atrophy: Phase I Ascending-Dose Study (STRONG)

Author

Finkel, Richard S., primary, Darras, Basil T., additional, Mendell, Jerry R., additional, Day, John W., additional, Kuntz, Nancy L., additional, Connolly, Anne M., additional, Zaidman, Craig M., additional, Crawford, Thomas O., additional, Butterfield, Russell J., additional, Shieh, Perry B., additional, Tennekoon, Gihan, additional, Brandsema, John F., additional, Iannaccone, Susan T., additional, Shoffner, John, additional, Kavanagh, Sarah, additional, Macek, Thomas A., additional, and Tauscher-Wisniewski, Sitra, additional

Published
2023
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5. Assessing Bulbar Function in Spinal Muscular Atrophy Using Patient-Reported Outcomes

Author

Dunaway Young, Sally, primary, Pasternak, Amy, additional, Duong, Tina, additional, McGrattan, Katlyn E., additional, Stranberg, Sarah, additional, Maczek, Elizabeth, additional, Dias, Courtney, additional, Tang, Whitney, additional, Parker, Dana, additional, Levine, Alexis, additional, Rohan, Alyssa, additional, Wolford, Connie, additional, Martens, William, additional, McDermott, Michael P., additional, Darras, Basil T., additional, and Day, John W., additional

Published
2023
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7. Psychometric properties of the PEDI-CAT for children and youth with spinal muscular atrophy

Author

Fragala-Pinkham, Maria, primary, Pasternak, Amy, additional, McDermott, Michael P., additional, Mirek, Elizabeth, additional, Glanzman, Allan M., additional, Montes, Jacqueline, additional, Dunaway Young, Sally, additional, Salazar, Rachel, additional, Quigley, Janet, additional, Riley, Susan O., additional, Chiriboga, Claudia A., additional, Finkel, Richard S., additional, Tennekoon, Gihan, additional, Martens, William B., additional, De Vivo, Darryl C., additional, and Darras, Basil T., additional

Published
2021
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9. Revised Recommendations for the Treatment of Infants Diagnosed with Spinal Muscular Atrophy Via Newborn Screening Who Have 4 Copies of SMN2

Author

Glascock, Jacqueline, primary, Sampson, Jacinda, additional, Connolly, Anne M., additional, Darras, Basil T., additional, Day, John W., additional, Finkel, Richard, additional, Howell, R. Rodney, additional, Klinger, Katherine W., additional, Kuntz, Nancy, additional, Prior, Thomas, additional, Shieh, Perry B., additional, Crawford, Thomas O., additional, Kerr, Douglas, additional, and Jarecki, Jill, additional

Published
2020
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10. Evaluator Training and Reliability for SMA Global Nusinersen Trials1

Author

Glanzman, Allan M., primary, Mazzone, Elena S., additional, Young, Sally Dunaway, additional, Gee, Richard, additional, Rose, Kristy, additional, Mayhew, Anna, additional, Nelson, Leslie, additional, Yun, Chris, additional, Alexander, Katie, additional, Darras, Basil T., additional, Zolkipli-Cunningham, Zarazuela, additional, Tennekoon, Gihan, additional, Day, John W., additional, Finkel, Richard S., additional, Mercuri, Eugenio, additional, De Vivo, Darryl C., additional, Baldwin, Ron, additional, Bishop, Kathie M., additional, and Montes, Jacqueline, additional

Published
2018
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11. Disease Trajectories in the Revised Hammersmith Scale in a Cohort of Untreated Patients with Spinal Muscular Atrophy types 2 and 3.

Author

Wolfe A, Stimpson G, Ramsey D, Coratti G, Dunaway Young S, Mayhew A, Pane M, Rohwer A, Muni Lofra R, Duong T, O'Reilly E, Milev E, Civitello M, Sansone VA, D'Amico A, Bertini E, Messina S, Bruno C, Albamonte E, Mazzone E, Main M, Montes J, Glanzman AM, Zolkipli-Cunningham Z, Pasternak A, Marini-Bettolo C, Day JW, Darras BT, De Vivo DC, Baranello G, Scoto M, Finkel RS, Mercuri E, and Muntoni F

Subjects
Humans, Female, Male, Child, Child, Preschool, Adolescent, Disease Progression, Cohort Studies, Severity of Illness Index, Longitudinal Studies, Scoliosis therapy, Scoliosis physiopathology, Spinal Fusion, Infant, Spinal Muscular Atrophies of Childhood physiopathology, Spinal Muscular Atrophies of Childhood therapy
Abstract

Background: Spinal muscular atrophy (SMA) is a neuromuscular disorder characterised by progressive motor function decline. Motor function is assessed using several functional outcome measures including the Revised Hammersmith Scale (RHS)., Objective: In this study, we present longitudinal trajectories for the RHS in an international cohort of 149 untreated paediatric SMA 2 and 3 patients (across 531 assessments collected between March 2015 and July 2019)., Methods: We contextualise these trajectories using both the Hammersmith Functional Motor Scale Expanded (HFMSE) and Revised Upper Limb Module (RULM). At baseline, this cohort included 50% females and 15% of patients had undergone spinal fusion surgery. Patient trajectories were modelled using a natural cubic spline with age, sex, and random effects for each patient., Results: RHS and HFMSE scores show similar trends over time in this cohort not receiving disease modifying therapies. The results confirm the strong correlation between the RHS and RULM previously observed in SMA types 2 and 3a. Scoliosis surgery is associated with a reduction of 3 points in the RHS, 4.5 points in the HFMSE for the SMA 2 population, and a reduction of 11.8 points in the RHS, and 13.4 points in the HFMSE for the SMA 3a populations. When comparing the RHS and RULM, there is a lower correlation in the type 3a's than the type 2 patients. In the SMA 2 population, there is no significant difference between the sexes in either the RHS or HFMSE trajectories. There is no significant difference in the RULM trajectory in the SMA 2 or 3a participants by sex., Conclusions: This study demonstrates that the RHS could be used in conjunction with other functional measures such as the RULM to holistically detect SMA disease progression. This will assist with fully understanding changes that occur with treatments, further defining trajectories and therapy outcomes.

Published
2024
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