Funding Portfolio

When we began our work, the level of investment in the Duchenne research field was nearly nonexistent. Over the years, Parent Project Muscular Dystrophy (PPMD) has acted as a catalyst — funding research that is both promising and cutting edge. We were the first organization to create a drug development initiative on validated targets (Project Catalyst) to invest in the generation of boys currently affected by Duchenne. As a result of our investment, the field is now ripe with promise.

The following chart shows the various research initiatives and the awards given to those trying to develop new treatments for this generation of young men.

Make sure to:

• Click on a research initiative in the chart to learn more
• Click on a column heading to sort
  
  

	Year	Title	Recipient	Type	Amount
	2011	Validation & Preclinical studies on Drugs discovered by Utrophin promoter HTS for DMD therapy	Dr. Tejvir Khurana, University of Pennsylvania	Research Grant	$97,869
	2011	Follistatin gene therapy to improve quadriceps muscle strength	Dr. Jerry Mendell, Nationwide Children's Hospital	Research Grant	$600,000
	2011	Prevention of eccentric stress damage and contractile dysfunction by glycoprotein-based membrane strengthening in cardiac muscle	Dr. Paul Janssen, Ohio State University	Research Grant	$50,000
	2011	Optimization of renin-angiotensin-aldosterone inhibitors as a treatment for dystrophin-deficient skeletal muscles and heart	Dr. Jill Rafael-Fortney, Ohio State University	Research Grant	$50,000
	2011	Analysis of protein interactions mediated by micro-dystrophin in the mdx heart	Dr. Federica Montanaro, Ohio State University	Bridge Grant	$50,000
	2011	Tadalafil/Sildenafil Study for muscle and heart function in DMD	Dr. Ron Victor, Cedars Sinai Medical Center	Research Grant	$753,625
	2004	Project Catalyst: New Drug Development	PTC Therapeutics	Research Grant	$2,500,000
	2010	Identify novel AAV vectors for canine heart transduction by peripheral vein injection	Dr. Dongsheng Duan, University of Missouri	Research Grant	$116,069
	2010	Failed Regeneration in the Muscular Dystrophies	Dr. Lee Sweeney, University of Pennsylvania	Research Grant	$1,050,000
	2011	Failed Regeneration in the Muscular Dystrophies	Dr. Stan Nelson, UCLA	Research Grant	$262,499
	2010	Acceleron ACE-031	Dr. Jean Mah, Alberta Children's Hospital	Clinical Trial Travel Funds	$5,000
	2010	Acceleron ACE-031	Dr. Mark Tarnopolsky, McMaster University	Clinical Trial Travel Funds	$5,000
	2010	Acceleron ACE-031	Dr. Craig Campbell, Lawson Health Research Institute	Clinical Trial Travel Funds	$5,000
	2010	Acceleron ACE-031	Dr. Katherine Selby, BC Children’s Hospital	Clinical Trial Travel Funds	$5,000
	2011	AVI-4658	Dr. Jerry Mendell, Nationwide Children's Research Institute	Clinical Trial Travel Funds	$50,000
	2011	A New Technology to Isolate Muscle Stem cells for Research	Nicholas Dobes	Research Grant	$50,000
	2011	FASEB Muscle Stem Cell Meeting	Dr. Thomas Rando	Meeting Support	$10,000
	2010	MD 2010 Conference	Dr. Steve Wilton, University of Western Australia, Perth	Meeting Support	$5,000
	2010	Antisense Oligonucleotides (AON) Therapies in Neuromuscular Disorders	Dr. Edward Connor, Children's National Medical Center	Meeting Support	$10,000
	2009	nNos as a potential therapy for DMD	Nicholas Whitehead, University of Washington	Research Grant	$50,000
	2009	Large animal model/studies Therapeutics in DMD	Dr. Meg Sleeper, University of Pennsylvania	Research Grant	$100,000
	2009	Osteopontin as a therapeutic target in DMD (anti-fibrotic)	Melissa Spencer, UCLA	Research Grant	$50,000
	2009	Improving the efficiency of antisense oligonucleotides (exon skipping)	Carmen Bertoni, UCLA	Research Grant	$75,000
	2009	Validate MRI as an outcome measure in DMD	Krista Vandenborne, U. Florida	Research Grant	$200,000
	2009	Lam III, Integrin as a potential therapy for DMD	Bradley Hodges, Prothelia	Research Grant	$46,000
	2008	Biglycan	Justin Fallon, Brown University	Research Grant	$289,000
	2009	Combined therapeutics as a treatment for DMD	H. Lee Sweeney, U. Pennsylvania	Research Grant	$300,000
	2008	6 minute walk primary outcome measure DMD	PTC Therapeutics, Craig McDonald	Research Grant	$100,000
	2010	Outcome measures for NON-AMBULATORY patients	PTC Therapeutics	Research Grant	$50,000
	2011	TREAT-NMD Advisory Committee for Therapeutics (TACT) 4th Meeting	Dr. Volker Straub, University of Newcastle upon Tyne	Meeting Support	$25,000
	2011	2nd Annual Clinical Outcome Measures in DMD Workshop	Dr. Abby Bronson, Children's National Medical Center	Meeting Support	$1,300
	2011	TREAT-NMD Advisory Committee for Therapeutics (TACT) 3rd Review Meeting	Dr. Volker Straub, University of Newcastle upon Tyne	Meeting Support	$24,577
	2011	Biomarker study in DMD	Somalogic, Inc and Cincinnati Children’s Hospital Medical Center	Research Grant	Up to $40,900
	2011	Therapeutic application of selective glucocorticoid receptor modulators in Duchenne muscular dystrophy - balancing efficacy with an improved side-effect profile	Dr. Tony Huynh, Children's Research Institute	Research Grant	$100,000
	2011	Clinical Development of SMTC1100, a Utrophin Upregulator for Duchenne Muscular Dystrophy	Dr. Jon Tinsley, Summit	Research Grant	$250,000
	2011	Review of Approved Drugs for Duchenne (RADD) Working Group	Children’s National Medical Center, Nationwide Children’s Research Institute, and TREAT-NMD	Research Grant	$100,000
	2011	Combinatorial Use of Sildenafil and Prednisone to Treat Dystrophin-Deficient Cardiac and Skeletal Muscle	Dr. Stan Froehner, University of Washington	Research Grant	$132,322
	2011	Prospective Assessment of the Prevalence of Autism and Obsessive Compulsive Disorder in the Iowa childhood onset Duchenne Becker Muscular Dystrophy Population	Dr. Katherine Mathews, University of Iowa Children’s Hospital	Research Grant	$33,337.50
	2012	Recombinant Biglycan for Treatment of Duchenne and Becker Muscular Dystrophy	Joel Braunstein, Tivorsan Pharmaceuticals	Research Grant	$565,000
	2011	Canadian DMD National Bone Health Research Initiative	Dr. Leanne Ward, Children’s Hospital of Eastern Ontario	Meeting Support	$37,000
	2012	Analysis of protein interactions mediated by micro-dystrophin in the mdx heart	Dr. Federica Montanaro, Ohio State University	Research Grant	$52,500
	2012	Pilot Project to identify genetic modifiers of Duchenne muscular dystrophy	Dr. Stanley Nelson, University of California, Los Angeles	Research Grant	$100,000
	2012	Exons 45-55 skipping with a cocktail antisense oligo	Dr. Toshifumi Yokota, University of Alberta	Research Grant	$50,000
	2012	Long-term efficacy and side effect profile of compound A, a selective glucocorticoid receptor modulator in mdx mice	Dr. Kanneboyina Nagaraju, Children’s Research Institute	Research Grant	$50,000
	2012	Development of MEMRI as an outcome measure for pre-clinical studies in the mdx mouse	Dr. Volker Straub, University of Newcastle Upon Tyne	Research Grant	$49,003
	2014	Predicting the benefit vs the side effects of corticosteroids in Duchenne muscular dystrophy	Dr. Robert Griggs, University of Rochester	Research Grant	$78,357
	2012	Magnetic Resonance Imaging as a Biomarker in Muscular Dystrophy Supplement	Dr. Kristen Vandenborne, University of Florida	Research Grant	$59,598
	2012	Clinically Meaningful Outcomes for Duchenne Muscular Dystrophy Therapeutic Trials	Dr. Erik Henricson, University of California, Davis	Research Grant	$175,000
	2012	Early treatment with Aldosterone antagonism attenuates cardiomyopathy in Duchenne MD (Epleronone Trial)	Dr. Kan Hor, Cincinnati Children's Hospital Medical Center	Research Grant	$84,092
	2012	Laminin-111 protein therapy for Duchenne Muscular Dystrophy	Dr. Dean Burkin, University of Nevada, Reno	Research Grant	$25,000
	2012	TREAT-NMD Advisory Committee for Therapeutics (TACT). 6th Meeting, Prague, Czech Republic. 27th-28th October 2012	Dr. Volker Straub, University of Newcastle Upon Tyne	Meeting Grant	$24,186
	2012	NCH/Wellstone/OSU Myology Course	Dr. Kevin Flanigan, Nationwide Children’s Hospital	Meeting Grant	$25,440
	2012	SERCA2a gene therapy for Duchenne cardiomyopathy	Dr. Donsheng Duan, University of Missouri	Research Grant	$280,425
	2012	Preclinical investigation of tamoxifen in mdx miceas prerequisite for a clinical trial in DMD patients	Dr. Urs Ruegg, University of Geneva	Research Grant	$164,100
	2012	TREAT-NMD Advisory Committee for Therapeutics (TACT). 5th Meeting, Arlington VA, USA. 28-29 April 2012	Dr. Volker Straub, University of Newcastle upon Tyne	Meeting Grant	$24,355
	2012	A Randomized, Double-blind, Placebo-controlled, Multiple-dose, Dose-escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetic, and Pharmacodynamic Effects of HT-100 in Patients with Duchenne Muscular Dystrophy	Marc Blaustein, Halo Therapeutics, LLC	GIFTED	$100,000

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