KevinMatthewFlaniganMD

Neuromuscular Medicine

Director, Center for Gene Therapy, and Robert F. & Edgar T. Wolfe Foundation Endowed Chair in Neuromuscular Research, Nationwide Children’s Hospital; Professor of Pediatrics and Neurology at the Ohio State University College of Medicine

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Office
700 Childrens Dr
Columbus, OH 43205
Phone+1 614-722-5615
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Education & Training

Johns Hopkins UniversityFellowship, Neuromuscular Medicine (Neurology), 1994 - 1995
Johns Hopkins UniversityResidency, Neurology, 1991 - 1994
University of Michigan Health SystemInternship, Internal Medicine, 1990 - 1991
Rush Medical College of Rush University Medical CenterClass of 1990

Certifications & Licensure

OH State Medical License 2010 - 2025
UT State Medical License 1995 - 2010
MI State Medical License 1990 - 1991
American Board of Psychiatry and Neurology Neurology
American Board of Psychiatry and Neurology Neuromuscular Medicine

Clinical Trials

Finding the Optimum Regimen for Duchenne Muscular Dystrophy Start of enrollment: 2013 Jan 01
Completed
Phase 3
A Study of the Safety, Tolerability & Efficacy of Long-term Administration of Drisapersen in US & Canadian Subjects Start of enrollment: 2013 May 01
Terminated
Phase 3
Natural History Studies of Mucopolysaccharidosis III Start of enrollment: 2014 Feb 01
Completed
Gene Transfer Clinical Trial for Duchenne Muscular Dystrophy Using rAAVrh74.MCK.GALGT2 Start of enrollment: 2016 Apr 01
Withdrawn
Phase 1, Phase 2
Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH Start of enrollment: 2016 Mar 01
Active, not recruiting
Phase 2, Phase 3
Gene Transfer Clinical Trial for Mucopolysaccharidosis (MPS) IIIB Start of enrollment: 2017 Oct 16
Terminated
Phase 1, Phase 2
Gene Transfer Clinical Trial to Deliver rAAVrh74.MCK.GALGT2 for Duchenne Muscular Dystrophy Start of enrollment: 2017 Nov 06
Active, not recruiting
Phase 1, Phase 2
Spironolactone Versus Prednisolone in DMD Start of enrollment: 2018 Dec 05
Terminated
Phase 1
A Long-term Follow-up Study of Patients With MPS IIIA Treated With ABO-102 Start of enrollment: 2020 Sep 28
Enrolling by invitation
Phase 3
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Publications & Presentations

PubMed

6 citations
A genome-wide association analysis of loss of ambulation in dystrophinopathy patients suggests multiple candidate modifiers of disease severity.
Kevin M Flanigan, Megan A Waldrop, Paul T Martin, Roxane Alles, Diane M Dunn, Lindsay N Alfano, Tabatha R Simmons, Melissa Moore-Clingenpeel, John Burian, Sang-Cheol S...> ;European Journal of Human Genetics. 2023 Jun 1
5 citations
Phenotypic Spectrum of Dystrophinopathy Due to Duchenne Muscular Dystrophy Exon 2 Duplications.
Alberto A Zambon, Megan A Waldrop, Roxane Alles, Robert B Weiss, Sara Conroy, Melissa Moore-Clingenpeel, Stefano Previtali, Kevin M Flanigan> ;Neurology. 2022 Feb 15
Automated immunofluorescence analysis for sensitive and precise dystrophin quantification in muscle biopsies.
Tatyana A. Vetter, Stefan Nicolau, Adrienne J Bradley, Emma C Frair, Kevin M. Flanigan> ;Neuropathology and Applied Neurobiology. 2021 Nov 30
5 citations
Validity and Reliability of the Neuromuscular Gross Motor Outcome.
Lindsay N. Alfano, M. Iammarino, Natalie F. Reash, Brenna R. Powers, Kiana Shannon, Anne M. Connolly, Megan A. Waldrop, Garey Noritz, Richard Shell, Chang-Yong Tsao, K...> ;Pediatric Neurology. 2021 Sep 1
2 citations
Longitudinal MRI brain volume changes over one year in children with mucopolysaccharidosis types IIIA and IIIB.
Nicolas J. Abreu, Bhavani Selvaraj, Kristen V. Truxal, Melissa Moore-Clingenpeel, Nicholas Zumberge, Kelly A. McNally, Kim L. McBride, Mai-Lan Ho, Kevin M. Flanigan> ;Molecular Genetics and Metabolism. 2021 Apr 27
22 citations
Update in the Mucopolysaccharidoses
Kim L. McBride, Kevin M. Flanigan> ;Seminars in Pediatric Neurology. 2021 Feb 10
24 citations
Lack of Toxicity in Nonhuman Primates Receiving Clinically Relevant Doses of an AAV9.U7snRNA Vector Designed to Induce DMD Exon 2 Skipping
Liubov V Gushchina, Emma C Frair, Natalie Rohan, Adrienne J Bradley, Tabatha R. Simmons, Hemantkumar D Chavan, Hsin-Jung Chou, Michelle Eggers, Megan A. Waldrop, Nicol...> ;Human Gene Therapy. 2021 May 7
5 citations
Gene editing and modulation for Duchenne muscular dystrophy.
Anthony A Stephenson, Kevin M. Flanigan> ;Progress in Molecular Biology and Translational Science. 2021 Jan 1
4 citations
Parent Project Muscular Dystrophy Females with Dystrophinopathy Conference, Orlando, Florida June 26 - June 27, 2019.
Susan D. Apkon, Kathi Kinnett, Linda H. Cripe, Dongsheng Duan, Jamie L. Jackson, Joe N. Kornegay, May Ling Mah, Stanley F. Nelson, Vamshi Rao, Mena Scavina, Brenda L. ...> ;Journal of Neuromuscular Diseases. 2021 Jan 1
12 citations
Natural History of Steroid-Treated Young Boys With Duchenne Muscular Dystrophy Using the NSAA, 100m, and Timed Functional Tests.
N. Miller, Lindsay N. Alfano, M. Iammarino, Anne M. Connolly, Melissa Moore-Clingenpeel, Brenna R. Powers, Chang-Yong Tsao, Megan A. Waldrop, Kevin M. Flanigan, Jerry ...> ;Pediatric Neurology. 2020 Dec 1
33 citations
Meta-analyses of ataluren randomized controlled trials in nonsense mutation Duchenne muscular dystrophy.
Craig Campbell, Richard J. Barohn, Enrico Bertini, Brigitte Chabrol, Giacomo P. Comi, Basil T. Darras, Richard S. Finkel, Kevin M. Flanigan, Nathalie Goemans, Susan T....> ;Journal of Comparative Effectiveness Research. 2020 Aug 27
11 citations
X-linked muscular dystrophy in a Labrador Retriever strain: phenotypic and molecular characterisation.
Inès Barthélémy, Nadège Calmels, Robert B. Weiss, Laurent Tiret, Adeline Vulin, Nicolas Wein, Cécile Peccate, Carole Drougard, Christophe Béroud, Nathalie Deburgrave, ...> ;Skeletal Muscle. 2020 Aug 7
16 citations
The Genotypic and Phenotypic Spectrum of BICD2 Variants in Spinal Muscular Atrophy.
Daniel C. Koboldt, Megan A. Waldrop, Richard K. Wilson, Kevin M. Flanigan> ;Annals of Neurology. 2020 Apr 1
5 citations
Homozygous variants in AMPD2 and COL11A1 lead to a complex phenotype of pontocerebellar hypoplasia type 9 and Stickler syndrome type 2
Nicolas J. Abreu, Daniel C. Koboldt, Julie M. Gastier-Foster, Ashita Dave-Wala, Kevin M. Flanigan, Megan A. Waldrop> ;American Journal of Medical Genetics. Part A. 2020 Mar 1
12 citations
Truncating variants in UBAP1 associated with childhood-onset nonsyndromic hereditary spastic paraplegia
Shen Gu, Chun-An Chen, Jill A. Rosenfeld, Heidi Cope, Nathalie Launay, Kevin M. Flanigan, Megan A. Waldrop, Rachel Schrader, Jane Juusola, Ozlem Goker-Alpan, Aubrey Mi...> ;Human Mutation. 2020 Mar 1
16 citations
Clinical Phenotypes of DMD Exon 51 Skip Equivalent Deletions: A Systematic Review.
Megan A. Waldrop, Rabah Ben Yaou, Karin K Lucas, Ann Martin, Erin O'Rourke, Filnemus, Alessandra Ferlini, Francesco Muntoni, Sylvie Tuffery-Giraud, Robert B. Weiss, Ke...> ;Journal of Neuromuscular Diseases. 2020 Jan 1
10 citations
Variants in MED12L, encoding a subunit of the mediator kinase module, are responsible for intellectual disability associated with transcriptional defect.
Mathilde Nizon, Vincent Laugel, Kevin M Flanigan, Matthew Pastore, Megan A Waldrop, Jill A Rosenfeld, Ronit Marom, Rui Xiao, Amanda Gerard, Olivier Pichon, Cédric Le C...> ;Genetics in Medicine. 2019 Dec 1
Correction: Variants in MED12L, encoding a subunit of the Mediator kinase module, are responsible for intellectual disability associated with transcriptional defect.
Mathilde Nizon, Vincent Laugel, Kevin M Flanigan, Matthew Pastore, Megan A Waldrop, Jill A Rosenfeld, Ronit Marom, Rui Xiao, Amanda Gerard, Olivier Pichon, Cédric Le C...> ;Genetics in Medicine. 2019 Nov 1
60 citations
Update in Duchenne and Becker muscular dystrophy.
Megan A. Waldrop, Kevin M. Flanigan> ;Current Opinion in Neurology. 2019 Oct 1
11 citations
Evaluation of biomarkers for Sanfilippo syndrome.
Jennifer T. Saville, Kevin M. Flanigan, Kristen V. Truxal, Kim L. McBride, Maria Fuller> ;Molecular Genetics and Metabolism. 2019 Sep 1
4 citations
Young Becker Muscular Dystrophy Patients Demonstrate Fibrosis Associated With Abnormal Left Ventricular Ejection Fraction on Cardiac Magnetic Resonance Imaging.
T. Pace Johnston, Linda H. Cripe, May Ling Mah, Melissa Moore-Clingenpeel, Cody Young, Kevin M. Flanigan, Jerry R. Mendell, Kan N Hor> ;Circulation. Cardiovascular Imaging. 2019 Jul 1
Twice weekly Glucocorticosteroids in Infants and Young boys with Duchenne Muscular Dystrophy.
Connolly, A. M.,Zaidman, C. M.,Golumbek, P. T.,Cradock, M. M.,Flanigan, K. M.,Kuntz, N. L.,Finkel, R. S.,McDonald, C. M.,Iannaccone, S. T.,Anand, P.,Siener, C. A.,Flor...> ;Muscle Nerve. 2019 Feb 02
25 citations
Diagnostic Utility of Whole Exome Sequencing in the Neuromuscular Clinic.
Megan A. Waldrop, Matthew Pastore, Rachel Schrader, Emily Sites, Dennis Bartholomew, Chang-Yong Tsao, Kevin M. Flanigan> ;Neuropediatrics. 2019 Jan 21
12 citations
In-frame de novo mutation in BICD2 in two patients with muscular atrophy and arthrogryposis.
Daniel C. Koboldt, Rama D. Kastury, Megan A. Waldrop, Benjamin J. Kelly, Theresa Mihalic Mosher, Heather M. McLaughlin, Don Corsmeier, Jonathan L. Slaughter, Kevin M. ...> ;Cold Spring Harbor Molecular Case Studies. 2018 Oct 1
12 citations
An Isolated Limb Infusion Method Allows for Broad Distribution of rAAVrh74.MCK.GALGT2 to Leg Skeletal Muscles in the Rhesus Macaque.
Rui Xu, Ying Jia, Deborah A. Zygmunt, Megan L. Cramer, Kelly E. Crowe, Guohong Shao, Agatha E. Maki, Haley N. Guggenheim, Benjamin C. Hood, Danielle A. Griffin, Ellyn ...> ;Molecular Therapy. Methods & Clinical Development. 2018 Jul 14
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Press Mentions

Can Gene Editing Kill Deadly Diseases?April 11th, 2023
Muscular Dystrophy Association Virtual Conference to Feature Latest Advances in Neuromuscular Therapy and Clinical CareMarch 8th, 2021
Abeona Therapeutics Announces Gene Therapy Clinical Trial and Preclinical Program Presentations at ASGCT 20th Annual MeetingMay 8th, 2017
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