Malan Syndrome Foundation – Research Grant Program

Malan Syndrome Foundation – Research Grant Program is sponsored by Malan Syndrome Foundation. This program supports basic, translational, and clinical research that aims to advance the current understanding of Malan syndrome and identify disease-modifying treatments.

RESEARCH GRANT PROGRAM | Malan The purpose of the research grant program is to advance the current understanding of Malan syndrome and identify disease-modifying treatments. Awards are for one- to two- years; scientific investigators at all stages of their career are encouraged to apply.

Grant awards are based on proposal evaluation by the Malan Syndrome Foundation Medical and Scientific Advisory Board with the approval of the Board of Directors. The Malan Syndrome Foundation supports collaborative efforts and the sharing of information to increase the knowledge base for Malan syndrome and promote further discovery.

Special areas of interest: Creating novel disease models that replicate the human phenotype Increase understanding of mechanisms regulating NFIX gene expression; identification of druggable targets that can increase NFIX expression and rescue haploinsufficient phenotype Identification of molecular pathophysiology associated with Malan syndrome in the central nervous system as well as in cardiovascular, orthopedic, neuro-ophthalmologic, gastrointestinal and other pertinent areas that can inform translational research for drug discovery The Research Grant Program Guidelines can be found here .

Full proposals (by invite only) can be submitted here .

Michael Piper, PhD, University of Queensland, Australia, Characterization of a humanized mouse model for Malan syndrome , $22,404 + an additional $75,000 provided by RTW Foundation , 2-year grant; Research summary available here Senyene Hunter, MD, PhD, University of North Carolina at Chapel Hill, Phenotyping epilepsy in individuals with Malan syndrome , $15,000, 1-year grant Jessica Cale, PhD, Murdoch University, Antisense oligonucleotide-mediated therapeutic intervention for Malan syndrome , $30,000, 1-year research grant (Year 2); Research summary available here InVivo Biosystems, Repurposed drug screen in a C.

elegans model of Malan syndrome, $140,250 Erin Heinzen, PharmD, PhD and Nate Hathaway, PhD, University of North Carolina at Chapel Hill, Using genome-wide CRISPR screens to identify regulators of NFIX expression , $30,000, 2-year research grant InVivo Biosystems, Characterization of a C.

elegans model of Malan syndrome and assay optimization for repurposed drug screening , $30,175 Kreepa Kooblall, PhD, University of Oxford, Investigating the pathological molecular mechanisms of Malan syndrome and identifying pharmacological agents for treatment , $30,000, 1-year research grant Craig McIntosh, PhD, Murdoch University, Antisense oligomer-mediated therapeutic intervention for Malan syndrome , $30,000, 1-year research grant (Year 1) Thomas Frazier, PhD, John Carroll University, Development and validation of a neurobehavioral evaluation tool , $14,200, 1-year research grant Kathryn Hixson, PhD, University of North Carolina at Chapel Hill, Elucidating the molecular and cellular pathologies in an iPSC model of Malan syndrome to identify transformative therapies for modifying disease progression , $10,000 + an additional $10,000 provided by Uplifting Athletes, 1-year research grant