Waldrop MA - Search Results

1

Parental Experiences with Newborn Screening and Gene Replacement Therapy for Spinal Muscular Atrophy.

Meyer AP, Connolly AM, Vannatta K, Hacker N, Hatfield A, Decipeda A, Parker P, Willoughby A, Waldrop MA. Meyer AP, et al. Among authors: waldrop ma. J Neuromuscul Dis. 2024;11(1):129-142. doi: 10.3233/JND-230082. J Neuromuscul Dis. 2024. PMID: 38160362 Free PMC article.

2

A Novel De Novo Heterozygous SCN4a Mutation Causing Congenital Myopathy, Myotonia and Multiple Congenital Anomalies.

Waldrop M, Amornvit J, Pierson CR, Boue DR, Sahenk Z. Waldrop M, et al. J Neuromuscul Dis. 2019;6(4):467-473. doi: 10.3233/JND-190425. J Neuromuscul Dis. 2019. PMID: 31609695

3

Clinical Phenotypes of DMD Exon 51 Skip Equivalent Deletions: A Systematic Review.

Waldrop MA, Yaou RB, Lucas KK, Martin AS, O'Rourke E; FILNEMUS; Ferlini A, Muntoni F, Leturcq F, Tuffery-Giraud S, Weiss RB, Flanigan KM. Waldrop MA, et al. J Neuromuscul Dis. 2020;7(3):217-229. doi: 10.3233/JND-200483. J Neuromuscul Dis. 2020. PMID: 32417793

4

Spinal Muscular Atrophy in the Treatment Era.

Waldrop MA, Elsheikh BH. Waldrop MA, et al. Neurol Clin. 2020 Aug;38(3):505-518. doi: 10.1016/j.ncl.2020.03.002. Neurol Clin. 2020. PMID: 32703464 Review.

5

Gene Therapy for Spinal Muscular Atrophy: Safety and Early Outcomes.

Waldrop MA, Karingada C, Storey MA, Powers B, Iammarino MA, Miller NF, Alfano LN, Noritz G, Rossman I, Ginsberg M, Mosher KA, Broomall E, Goldstein J, Bass N, Lowes LP, Tsao CY, Mendell JR, Connolly AM. Waldrop MA, et al. Pediatrics. 2020 Sep;146(3):e20200729. doi: 10.1542/peds.2020-0729. Pediatrics. 2020. PMID: 32843442

6

Overview of gene therapy in spinal muscular atrophy and Duchenne muscular dystrophy.

Abreu NJ, Waldrop MA. Abreu NJ, et al. Among authors: waldrop ma. Pediatr Pulmonol. 2021 Apr;56(4):710-720. doi: 10.1002/ppul.25055. Epub 2020 Sep 14. Pediatr Pulmonol. 2021. PMID: 32886442 Review.

7

Natural History of Steroid-Treated Young Boys With Duchenne Muscular Dystrophy Using the NSAA, 100m, and Timed Functional Tests.

Miller NF, Alfano LN, Iammarino MA, Connolly AM, Moore-Clingenpeel M, Powers BR, Tsao CY, Waldrop MA, Flanigan KM, Mendell JR, Lowes LP. Miller NF, et al. Among authors: waldrop ma. Pediatr Neurol. 2020 Dec;113:15-20. doi: 10.1016/j.pediatrneurol.2020.08.013. Epub 2020 Aug 27. Pediatr Neurol. 2020. PMID: 32979653

8

Spinal Muscular Atrophy.

Nicolau S, Waldrop MA, Connolly AM, Mendell JR. Nicolau S, et al. Among authors: waldrop ma. Semin Pediatr Neurol. 2021 Apr;37:100878. doi: 10.1016/j.spen.2021.100878. Epub 2021 Feb 11. Semin Pediatr Neurol. 2021. PMID: 33892848 Review.

9

Time is muscle: A recommendation for early treatment for preterm infants with spinal muscular atrophy.

Lee BH, Waldrop MA, Connolly AM, Ciafaloni E. Lee BH, et al. Among authors: waldrop ma. Muscle Nerve. 2021 Aug;64(2):153-155. doi: 10.1002/mus.27261. Epub 2021 May 26. Muscle Nerve. 2021. PMID: 33959970

10

Validity and Reliability of the Neuromuscular Gross Motor Outcome.

Alfano LN, Iammarino MA, Reash NF, Powers BR, Shannon K, Connolly AM, Waldrop MA, Noritz GH, Shell R, Tsao CY, Flanigan KM, Mendell JR, Lowes LP. Alfano LN, et al. Among authors: waldrop ma. Pediatr Neurol. 2021 Sep;122:21-26. doi: 10.1016/j.pediatrneurol.2021.05.021. Epub 2021 Jun 8. Pediatr Neurol. 2021. PMID: 34271497

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