HHS Recovery Act Recipient Reporting Readiness Tool
Step 4. Review and Copy the Grant Awards Data
TAGGS provides some – but not all – of the data needed for the Recipient Report. Recipients are responsible for directly collecting and reporting all required data to FederalReporting.gov. Data that HHS does not currently collect are highlighted in yellow. Do not copy this highlighted information. Please enter the appropriate data for your organization in these required fields. For assistance with entering these data please contact FederalReporting.gov.
You may capture the data HHS does provide by copying data from this screen and pasting it into the reporting format of your choice, such as the Excel spreadsheet template, the XML template, or by logging into the online form. For assistance with copying and pasting these data please e-mail our help desk at Readiness Help.
| Recipient Report: Grant or Loan | ||
| Prime Recipient |
| Reporting Information | ||
| Award Type | Award Number | Final Report |
| Grant | 1R21NS065936-01 | Recipient responsible for this data |
| Award Recipient Information | ||
| Recipient DUNS Number | Recipient Account Number | Recipient Congressional District |
| 042250712 | Recipient responsible for this data | 1 |
| Award Information | ||
| Funding Agency Code | Awarding Agency Code | Award Date |
| 7529 | 7529 | 07-14-2009 |
| Amount of Award | Sub Account Number for Program Source (TAS) | |
| $ 275,625 | Recipient responsible for this data | |
| Program Source (TAS)* | CFDA Number | |
| 750901 | 93.701 | |
| Total Number of Sub Awards to Individuals | Total Amount of Sub Awards to Individuals | |
| Recipient responsible for this data | Recipient responsible for this data | |
| Total Number of Payments to Vendors less than $25,000/award | Total Amount of Payments to Vendors less than $25,000/award | |
| Recipient responsible for this data | Recipient responsible for this data | |
| Total Number of Sub Awards less than $25,000/award | Total Amount of Sub Awards less than $25,000/award | |
| Recipient responsible for this data | Recipient responsible for this data | |
| Award Description | ||
| DESCRIPTION (provided by applicant): The long term goals of this proposal are to facilitate preclinical development of our novel therapeutic strategy for the currently incurable Limb Girdle Muscular Dystrophy Type 2B (LGMD2B). The muscular dystrophies are muscle wasting disorders with increased morbidity and mortality. LGMD2B is caused by dysferlin gene mutations leading to altered protein expression (dysferlinopathy). Deficient repair of damaged membranes via dysferlin-dependent membrane fusion has been implicated. However, lacunae exist in our knowledge of whether dysferlin regulates well-known vesicle fusion related processes (e.g. Acetylcholine Receptor (AChR) recycling & maintenance of synaptic transmission at the neuromuscular junction-NMJ). Using powerful pharmacogenetic tools available in the C. elegans model of LGMD2B, we have identified a novel role for fer-1 in the regulation of NMJ synaptic transmission (Preliminary studies). Our identification of a synaptopathic or myasthenic component has tremendous nosological and translational implications. Interestingly, Acetylcholine Esterase Inhibitors (AChEI) administration improves the survival of the A/J mouse model of LGMD2B in a small pilot trial (Preliminary Studies) and suggests the hypothesis that the NMJ deficit caused by dysferlin deficiency is amenable to treatment using AChEI as a candidate therapeutic to increase the efficiency of neuromuscular transmission and improve LGMD2B outcomes. As a specific aim we propose a well-designed, proof-of-principle study in order to complete the preliminary steps required for successful preclinical development of this candidate therapeutic and test our translational hypothesis. We will undertake a therapeutic trial where we treat 8 month old A/J mice with AChEI for 8 months. Therapeutic potential will be will be assayed using in vivo and ex vivo means to determine improvement of the dystrophic phenotype. The relevance and health relatedness of this project is that undertaking these studies we will ensure clearer understanding of the molecular and functional patho-physiology as well as facilitate preclinical development of our novel therapeutic strategy for LGMD2B. PUBLIC HEALTH RELEVANCE: The health relatedness of this project is that by completion of our aim we will ensure clearer understanding of the molecular and functional patho-physiology of limb-girdle muscular dystrophy Type 2B (LGMD2B) as well as facilitate preclinical development of our novel therapeutic strategy for LGMD2B. | ||
| Project Information | ||||||||||||||||||||||||||
| Project Name or Project/Program Title |
Project Status | Total Federal Amount ARRA Funds Received/Invoiced |
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| PRECLINICAL DEVELOPMENT OF A NOVEL THERAPEUTIC STRATEGY FOR LGMD2B | Recipient responsible for this data | Recipient responsible for this data | ||||||||||||||||||||||||
| Number of Jobs | Description of Jobs Created | |||||||||||||||||||||||||
| Recipient responsible for this data | Recipient responsible for this data | |||||||||||||||||||||||||
| Quarterly Activities/Project Description | ||||||||||||||||||||||||||
| Recipient responsible for this data | ||||||||||||||||||||||||||
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| Total Federal Amount of ARRA Expenditure |
Total Federal ARRA Infrastructure Expenditure |
Infrastructure Contact Name | ||||||||||||||||||||||||
| Recipient responsible for this data | Recipient responsible for this data | Recipient responsible for this data | ||||||||||||||||||||||||
| Infrastructure Contact Email | Infrastructure Contact Phone | Infrastructure Contact Phone Ext. | ||||||||||||||||||||||||
| Recipient responsible for this data | Recipient responsible for this data | Recipient responsible for this data | ||||||||||||||||||||||||
| Infrastructure Contact Street Address 1 | Infrastructure Contact Street Address 2 | Infrastructure Contact Street Address 3 | ||||||||||||||||||||||||
| 3451 WALNUT STREET | Not Available | Recipient responsible for this data | ||||||||||||||||||||||||
| Infrastructure City | Infrastructure State | Infrastructure ZIP Code+4 | ||||||||||||||||||||||||
| PHILADELPHIA | PA | 19104-6205 | ||||||||||||||||||||||||
| Infrastructure Purpose and Rationale | ||||||||||||||||||||||||||
| Recipient responsible for this data | ||||||||||||||||||||||||||
| Primary Place of Performance | ||
| Street Address 1 | Street Address 2 | City |
| 3700 HAMILTON WALK - D400 | Recipient responsible for this data | PHILADELPHIA |
| State | Zip Code+4 | Congressional District |
| PA | 191046205 | 2 |
| Country | ||
| US | ||
| Recipient Highly Compensated Officers | |||
| Prime Recipient Indication of Reporting Applicability | # | Officer Name | Officer Compensation |
| Recipient responsible for this data | 1 | Recipient responsible for this data | Recipient responsible for this data |
| 2 | Recipient responsible for this data | Recipient responsible for this data | |
| 3 | Recipient responsible for this data | Recipient responsible for this data | |
| 4 | Recipient responsible for this data | Recipient responsible for this data | |
| 5 | Recipient responsible for this data | Recipient responsible for this data | |
This concludes the current search.
To begin a new search, return to the HHS Recovery Act Recipient Reporting Readiness Tool.
USE IN THE RECIPIENT REPORT
The information provided by this tool is baseline data that the Recipient should include in the Recipient Report that must be submitted to FederalReporting.gov beginning October 1, 2009. The data from this tool can be cut and pasted directly into the Recipient Report.







