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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.

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Award Detail for: PRECLINICAL DEVELOPMENT OF A NOVEL THERAPEUTIC STRATEGY FOR LGMD2B
UNIVERSITY OF PENNSYLVANIA
DUNS Number: 042250712
3451 WALNUT STREET
PHILADELPHIA, PA 19104-6205
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
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
 
Activity Code (NAICS or NTEE-NPC)
1Recipient responsible for this data2Recipient responsible for this data
3Recipient responsible for this data4Recipient responsible for this data
5Recipient responsible for this data6Recipient responsible for this data
7Recipient responsible for this data8Recipient responsible for this data
9Recipient responsible for this data10Recipient responsible for this data
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

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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.