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Awarded Projects

Below is a list of ARM awarded projects.

1998-1999

PI Institution Project Description Amount Funding Type
Dr. Richard Gatti UCLA Patient identification and sample collection. $10,000 Gift

1999-2000

PI Institution Project Description Amount Funding Type
Dr. Zohar Argov Hadassah, Jerusalem Genotyping and sequencing efforts. $59,000 Gift
Dr. Richard Gatti UCLA Patient identification and linkage analysis. $2,000 Gift

2000-2001

PI Institution Project Description Amount Funding Type
Dr. Zohar Argov Hadassah, Jerusalem Genotyping and sequencing efforts. $52,000 Gift
Dr. Arastoo Vojdani Molecular Genetic Lab Sample collection and genotyping. $50,000 Gift
Dr. Richard Gatti UCLA HIBM, Linkage Analysis. $44,500 Gift
Dr. Chaim Jacob USC Sample collection and genotyping. $19,826 Gift

2001-2002

PI Institution Project Description Amount Funding Type
Dr. Richard Gatti UCLA Linkage analysis, HIBM. $17,760 Gift
Dr. Daniel Darvish HIBM Research Group Laboratory startup and sequencing. $30,000 Contract

2002-2003

PI Institution Project Description Amount Funding Type
Dr. Stella Mitrani-Rosenbaum Hadassah, Jerusalem Mechanism of pathogenesis of GNE/MNK in HIBM. $120,000 Grant 006
Dr. Hudson Freeze UCSD Initial Analysis and Potential Therapy for HIBM. $55,806 Grant 002
Dr. Valerie Askanas USC h-IBM Research. $55,000 Gift
Dr. Jianping Zheng USC Initial Study of antisense GNE/MNK and Over Expression of Mutant GNE in Human Skeletal Muscle Primary Culture System. $40,535 Grant 003

2003-2004

PI Institution Project Description Amount Funding Type
Dr. Jacques Tremblay Laval University, Canada Transplantation of genetically modified myoblasts from patients with IBM2. $73,000 Grant 008
Dr. Daniel Darvish HIBM Research Group Establishment of Cell Culture & Distribution Center for HIBM Research Samples and Reagents. $56,331 Grant 010
Dr.Kevin Yarema Johns Hopkins University Probing the Role of GNE/MNK Mutations in HIBM. $77,159 Grant 005

2004-2005

PI Institution Project Description Amount Funding Type
Dr. Jacques Tremblay Laval University, Canada Preparation of a clinical trial of transplantation of genetically modified myoblasts to HIBM patients. $92,100 Grant 015
Dr. Kevin Yarema Johns Hopkins University Probing the Role of GNE/MNK Mutations in HIBM (renewal). $111,872 Grant 011
Dr. Yadira Valles-Ayoub HIBM Research Group HIBM testing, and providing reagents and samples, and development of HIBM mouse model. $261,240 Contract

2005-2006

PI Institution Project Description Amount Funding Type
Dr. Jacques Tremblay Laval University, Canada Preparation of a clinical trial of transplantation of genetically modified myoblasts to HIBM patients. $77,159 Grant 015
Dr. Jon Wolff University of Wisconsin Preliminary Vector Construction and Validation for HIBM Gene Therapy. $15,000 Grant 016
Dr. Masashi Kitazawa UCI Impact of Amyloid-beta (Aß) overproduction on a mouse model of Hereditary IBM; Generation of Amyloid Precursor Protein (APP) & GNE double transgenic model. $45,000 Grant 013
Dr. Stella Mitrani-Rosenbaum Hadassah, Jerusalem Establishment of in-vitro and in-vivo experimental systems for Hereditary Inclusion Body Myopathy. $150,000 Grant 017
Dr. Yadira Valles-Ayoub HIBM Research Group HIBM testing, maintaining/expanding mouse colony, supporting researchers with samples and reagents $319,415 Contract

2006-2007

PI Institution Project Description Amount Funding Type
Dr. Ajit Varki UCSD Engineering Human-Like Sialic Acids in Mice with HIBM. $81,201 Grant 018
Dr. Jon Wolff University of Wisconsin Gene Therapy for Inclusion Body Myopathy Type II (IBM2). $100,000 Grant 019
Dr. Yadira Valles-Ayoub HIBM Research Group Laboratory Costs, HIBM testing, maintaining/expanding mouse colony, supporting researchers with samples and reagents $363,894 Contract
Dr. John Nemunaitis Mary Crowley Medical Research Center, TX Developing Gene Therapy for HIBM $50,000 Gift
Dr. Ichizo Nishino National Institute of Neurology, Japan Distal Myopathy with Rimmed Vacuoles (DMRV) & HIBM, Mouse Models $45,000 Gift

2007-2008

PI Institution Project Description Amount Funding Type
Dr. Yadira Valles-Ayoub HIBM Research Group Maintaining/expanding mouse colony, improvement of HIBM mouse model, maintaining CLIA and CAP accreditation, and developing Middle-Eastern genetic testing panel $218,046 Contract
Dr. Moises Mallo Gulbenkian Science Institute Identifying the critical source of sialic acid as a product of GNE activity $45,000 Grant 024

2008-2010

PI Institution Project Description Amount Funding Type
Dr. Yadira Valles-Ayoub HIBM Research Group Maintaining/expanding mouse colony, improvement of HIBM mouse model, developing and validating HIBM (GNE) gene therapy vector, GNE gene therapy toxicology analysis, maintaining CLIA and CAP accreditation, and developing Middle-Eastern genetic testing panel $107,192 Contract
Dr.Perry Shieh UCLA Fellowship Award for HIBM (Dr. Shirley Chi) $65,000 Fellowship Award

2010-2011

PI Institution Project Description Amount Funding Type
Dr. Yadira Valles-Ayoub HIBM Research Group Phenotype analysis of HIBM mouse model, developing and validating HIBM (GNE) gene therapy vector, in-vitro GNE gene therapy toxicology analysis, obtaining orphan drug designation, maintaining CLIA and CAP accreditation $214,000 Grant

2011-2012

PI Institution Project Description Amount Funding Type
Dr. Yadira Valles-Ayoub HIBM Research Group In-vivo GNE gene therapy toxicology analysis, maintaining CLIA and CAP accreditation, developing Middle-Eastern genetic testing panel $92,414 Grant

2012-2013

PI Institution Project Description Amount Funding Type
Dr. Yadira Valles-Ayoub HIBM Research Group Continuing pre-clinical toxicology studies, File Investigational New Drug (IND), maintaining mouse colony, maintaining CLIA and CAP accreditation, and launching Middle-Eastern genetic testing panel $185,160 Grant

2013-2014

PI Institution Project Description Amount Funding Type
Dr. Yadira Valles-Ayoub HIBM Research Group In-vivo GNE gene therapy toxicology analysis, Maintaining CLIA and CAP accreditation, Maintaining mouse colony, Continuing Middle-Eastern genetic testing Panel. $193,552 Grant

2014-2015

PI Institution Project Description Amount Funding Type
Dr. Yadira Valles-Ayoub HIBM Research Group Expanding and continuing Middle-Eastern genetic testing Panel, Maintaining CLIA and CAP accreditation, Maintaining mouse colony. $37,068 Grant