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ZEBRAFISH MUSCULAR DYSTROPHY MODEL FOR DRUG SCREENING

Award Number: R43AR055390
ORGANIZATION: NATIONAL INSTITUTE OF ARTHRITIS & MUSCULOSKELETAL & SKIN DISEASES
OPDIV: NIH
AWARD CLASS: DISCRETIONARY
AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)
PERIOD OF PERFORMANCE START DATE: 08/17/2007
PERIOD OF PERFORMANCE END DATE: 05/31/2008

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ZEBRAFISH MUSCULAR DYSTROPHY MODEL FOR DRUG SCREENING - DESCRIPTION (provided by applicant): This SBIR proposal aims to develop and validate a zebrafish model for screening drugs that mitigate symptoms associated with muscular dystrophy (MD). MD is comprised of several clinically and genetically heterogeneous disorders characterized by progressive weakness and degeneration of the skeletal or voluntary muscles which control movement. In some forms of MD, heart muscles and some involuntary muscles are also affected. MD can occur at any age. Duchenne is the most common form of MD affecting children, whereas myotonic is the most common form affecting adults. Although there has been tremendous improvement in the understanding of the molecular basis of MD, no effective treatment is currently available. Development of therapeutic strategies is a major focus of the biopharmaceutical industry. Murine and canine models have been useful in elucidating MD disease mechanisms and assessing potential therapeutic strategies, but neither model can be used for large scale genetic screens or for rapid drug screening. Although simpler in vivo model systems such as C. elegans (dys-1) have been developed, these invertebrate models are relatively poor predictors of efficacy and toxicity in humans because their metabolism and physiology are very different from those of humans. A new animal model that combines the advantages of invertebrate and mouse systems is needed to streamline the drug discovery process. This zebrafish model will facilitate screening of potential therapeutics that mitigate symptoms associated with muscular dystrophy and streamline the drug development timeline.


Issue Date FY	Funding FY	Legal Entity Name	Legal Entity Address	Legal Entity City	Legal Entity State	Legal Entity Zip Code	Legal Entity COUNTY	Legal Entity COUNTRY	Assistance Listing	Award Code	Budget Year	Action Date	Action Type	Action Amount

Issue Date FY: 2007 ( Subtotal = $161,810 )
2007	2007	PHYLONIX PHARMACEUTICALS, INC	100 INMAN STREET SUITE 300	CAMBRIDGE	MA	02139	MIDDLESEX	USA	Arthritis, Musculoskeletal and Skin Diseases Research	000	1	8/17/2007	NEW	$161,810
														Subtotal = $161,810

Grand Total All Awards = $161,810

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ZEBRAFISH MUSCULAR DYSTROPHY MODEL FOR DRUG SCREENING

Award Number: R43AR055390

ORGANIZATION: NATIONAL INSTITUTE OF ARTHRITIS & MUSCULOSKELETAL & SKIN DISEASES

OPDIV: NIH

AWARD CLASS: DISCRETIONARY

AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)

PERIOD OF PERFORMANCE START DATE: 08/17/2007

PERIOD OF PERFORMANCE END DATE: 05/31/2008

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