Genila Bibat, M.D.'s picture
Clinical Research Director, Center for Genetic Muscle Disorders
Phone: 443-923-2697
Kennedy Krieger Institute

707 N. Broadway
Baltimore, MD 21205
United States

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Dr. Bibat is a research scientist and the clinical research director for the Center for Genetic Muscle Disorders at Kennedy Krieger Institute. She is a research associate in Pediatrics at the Johns Hopkins University School of Medicine.

Dr. Bibat also serves as the sIRB liaison and Regulatory specialist for the Office of Human Research Administration (OHRA) at Kennedy Krieger.


Dr. Bibat received her Bachelor of Science in Psychology and Doctor of Medicine degrees at the University of Santo Tomas in Manila, Philippines. After completing her pediatric residency training at the University of Florida, Sacred Heart Children's Hospital, she became a Clinical Fellow in Neurodevelopmental Disabilities at the Kennedy Krieger Institute/Johns Hopkins School of Medicine from 1997 to 2000. She then became a research fellow in the Neurogenetics Department and subsequently joined the Institute as a Research Associate in March 2001.


Dr. Bibat has devoted her research effort in Neurogenetics to understand the neurobiological basis of Rett syndrome (RS), a neurodevelopemental disorder predominantly affecting females that is characterized by loss of developmental skills, primarily expressive language and purposeful hand use. She was involved in the NIH-funded RS Natural History Study as well as the FDA-funded Dextromethorphan (DM) drug trial in RS. In her work with other neurodegenerative disorders, particularly those with abnormalities of the white matter of the brain, Dr. Bibat has studied unclassified leukoencephalopathies. She has helped in the identification of new categories of disorders in which genetic studies are being pursued.

At the Center for Genetic Muscle Disorders, Dr. Bibat conducts extensive research, oversees clinical trials, and performs critical work in the field of neuromuscular disorders. Her initial work at the Center was with the NIH-funded FacioScapuloHumeral muscular dystrophy (FSHD) Wellstone research study. She has participated in the research studies under the FSHD Clinical Trial Network. She continues to work on multicenter industry sponsored clinical trials for neuromuscular disorders including FSHD, Duchenne and Becker Muscular Dystrophies, Myotonic Dystrophy, and Limb Girdle Muscular Dystrophy Type 1.

Dr. Bibat also serves as the Institute’s Single Institutional Review Board (sIRB) Liaison at the Office of Human Research Administration. She is responsible for sIRB studies and managing the reliance process and relationships between the Institute and external IRBs in the country. She also provides her expertise in the implementation of clinical research activities at the Institute, with an emphasis on industry-sponsored clinical trials and multi-center studies.

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Research Publications

The Clinical Development of Taldefgrobep Alfa: An Anti-Myostatin Adnectin for the Treatment of Duchenne Muscular Dystrophy.
Muntoni F, Byrne BJ, McMillan HJ, Ryan MM, Wong BL, Dukart J, Bansal A, Cosson V, Dreghici R, Guridi M, Rabbia M, Staunton H, Tirucherai GS, Yen K, Yuan X, Wagner KR; Taldefgrobep Alfa Study Group.Neurol Ther. 2024 Feb;13(1):183-219. doi: 10.1007/s40120-023-00570-w. Epub 2024 Jan 8.PMID: 38190001 

A phase Ib/IIa, open-label, multiple ascending-dose trial of domagrozumab in fukutin-related protein limb-girdle muscular dystrophy.
Leung DG, Bocchieri AE, Ahlawat S, Jacobs MA, Parekh VS, Braverman V, Summerton K, Mansour J, Stinson N, Bibat G, Morris C, Marraffino S, Wagner KR.Muscle Nerve. 2021 Aug;64(2):172-179. doi: 10.1002/mus.27259. Epub 2021 May 24.PMID: 33961310 Clinical Trial.

Longitudinal functional and imaging outcome measures in FKRP limb-girdle muscular dystrophy.
Leung DG, Bocchieri AE, Ahlawat S, Jacobs MA, Parekh VS, Braverman V, Summerton K, Mansour J, Bibat G, Morris C, Marraffino S, Wagner KR.BMC Neurol. 2020 May 19;20(1):196. doi: 10.1186/s12883-020-01774-5.PMID: 32429923 

Clinical trial readiness to solve barriers to drug development in FSHD (ReSolve): protocol of a large, international, multi-center prospective study.
LoRusso S, Johnson NE, McDermott MP, Eichinger K, Butterfield RJ, Carraro E, Higgs K, Lewis L, Mul K, Sacconi S, Sansone VA, Shieh P, van Engelen B, Wagner K, Wang L, Statland JM, Tawil R; ReSolve Investigators and the FSHD CTRN18.BMC Neurol. 2019 Sep 10;19(1):224. doi: 10.1186/s12883-019-1452-x.PMID: 31506080 

The NIH Toolbox for cognitive surveillance in Duchenne muscular dystrophy.
Thangarajh M, Kaat AJ, Bibat G, Mansour J, Summerton K, Gioia A, Berger C, Hardy KK, Wagner KR.Ann Clin Transl Neurol. 2019 Sep;6(9):1696-1706. doi: 10.1002/acn3.50867. Epub 2019 Aug 31.PMID: 31472009 

Are dopamine receptor and transporter changes in Rett syndrome reflected in Mecp2-deficient mice?
Wong DF, Blue ME, Brašić JR, Nandi A, Valentine H, Stansfield KH, Rousset O, Bibat G, Yablonski ME, Johnston MV, Gjedde A, Naidu S.Exp Neurol. 2018 Sep;307:74-81. doi: 10.1016/j.expneurol.2018.05.019. Epub 2018 May 18.PMID: 29782864

Randomized open-label trial of dextromethorphan in Rett syndrome.
Smith-Hicks CL, Gupta S, Ewen JB, Hong M, Kratz L, Kelley R, Tierney E, Vaurio R, Bibat G, Sanyal A, Yenokyan G, Brereton N, Johnston MV, Naidu S.Neurology. 2017 Oct 17;89(16):1684-1690. doi: 10.1212/WNL.0000000000004515. Epub 2017 Sep 20.PMID: 28931647 

Evaluation of QTc in Rett syndrome: Correlation with age, severity, and genotype.
Crosson J, Srivastava S, Bibat GM, Gupta S, Kantipuly A, Smith-Hicks C, Myers SM, Sanyal A, Yenokyan G, Brenner J, Naidu SR.Am J Med Genet A. 2017 Jun;173(6):1495-1501. doi: 10.1002/ajmg.a.38191. Epub 2017 Apr 10.PMID: 28394409 

Bone health in facioscapulohumeral muscular dystrophy: A cross-sectional study.
Chagarlamudi H, Corbett A, Stoll M, Bibat G, Grosmann C, Matichak Stock C, Stinson N, Shapiro J, Wagner KR.Muscle Nerve. 2017 Dec;56(6):1108-1113. doi: 10.1002/mus.25619. Epub 2017 Jun 5.PMID: 28214289

Functional outcomes in Rett syndrome.
Pidcock FS, Salorio C, Bibat G, Swain J, Scheller J, Shore W, Naidu S.Brain Dev. 2016 Jan;38(1):76-81. doi: 10.1016/j.braindev.2015.06.005. Epub 2015 Jul 11.PMID: 26175308