Below is a list of ARM awarded projects.
| PI | Institution | Project Description | Amount | Funding Type |
|---|---|---|---|---|
| Dr. Richard Gatti | UCLA | Patient identification and sample collection. | $10,000 | Gift |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |