This page is dedicated to the scientists who are pushing CMD basic science towards translational targets and helping Cure CMD fulfill its mission to identify drugs to slow disease progression.
Cure CMD Research Grants
Cure CMD Research Grant Goals
Cure CMD research goals reflect our mission to bring research, treatments and in the future, a cure for the congenital muscular dystrophies.
- Identify new therapeutic targets in the CMDs
- Identify small molecule compounds that work at these therapeutic target sites with priority towards drugs with existing pediatric FDA approval
- Facilitate the drug development process to ensure that each positive drug identification with promising preliminary data can be brought rapidly to preclinical trials and subsequent treatment trials
- Support and endorse clinical research, infrastructure and biomarker discovery required to launch effective and efficient clinical trials in rare disease
- Advance drug discovery with a diverse treatment pipeline
- Support new scientific investigators with innovative ideas and a commitment to CMD research
Struggle Against Muscular Dystrophy (S.A.M) CMD Translational Research Grant: Annual Deadline, Sept 15th.
S.A.M is a unique CMD advocacy group launched by two parents whose son has CMD. SAM’s focus is to support and foster CMD research through successful fundraising initiatives. The SAM Translational Research Award delivers upon its promise to target research focused on CMD treatment. The grant is made possible through Cure CMD and SAM collaboration and the Cure CMD research grant funding mechanism.
Cure CMD and SAM invite proposals to award a 1-2 year grant focusing on pre-clinical and clinical investigations that will advance treatment for congenital muscular dystrophy (CMD). The total award is limited to $100,000 (direct costs only). Annual RFA deadline Sept 15th. Grant application above. On the First Page of the Cure CMD grant application, please specify S.A.M. CMD Translational Research Grant
Cure CMD General Research Grants: Annual Deadline Sept 15th.
Elisabeth Giauque Short Term CMD Research Grant: Cure CMD provides a short-range or “seed” grant (usually $25,000- $50,000 per year for 1-2 years, direct costs only) to attract new investigators to the CMD field and assist existing investigators to support early phases of their research. The short-range grant focus is to collect preliminary data and test initial hypotheses. The short-range grant goal is to translate successful preliminary research to either second tier Cure CMD midrange grants (R-21 type) or third tier, long range (R-01 grants, 2-5 years) from outside funding sources, such as the National Institutes of Health. Annual RFA deadline, Sept 15th. Grant application above. On the first page of the Cure CMD grant application, please specify Elisabeth Giauque Short Term CMD Research Grant.
Past Awards:
2009 Cure CMD and S.A.M. Partner to Award over $200,000 in CMD research grants, click here
Cure CMD Postdoctoral and Clinician in Training Application for “Therapeutic Targets in the CMDs” conference, July 9-11, 2009 Click here to see the winners!
Upcoming and Past CMD and Muscular dystrophy Scientific and Medical Conferences
- World Muscle Society, Oct 12th-16th, 2010, Kumamoto, Japan
- Ottawa Conference on New Directions in Biology and Disease of Skeletal Muscle, May 5-8th, 2010Ottawa, Ontario, Canada, http://www.med.upenn.edu/muscle/registration.shtml
- CMD ENMC Outcome Measures workshop, March 5th-7th, 2010, Naarden, Holland
- CMD Drug Discovery Meeting, January 26th, 2010, National Chemical Center Genomics
- “Bringing down the Barriers- Translational Medicine in Inherited Neuromuscular Diseases” TREAT-NMD/NIH conference, Nov 17-19, 2009, Crowne Plaza Brussels City Centre, Brussels, Belgium (See: http://www.treat-nmd.eu/patients/events/events/401/ for more information)
- CMD Standard of Care Workshop, Nov 14th-16th, Brussels, Belgium
- Child Neurology Society, Oct 14-17, 2009, Louisville, Kentucky
- World Muscle Society, Sept 9-12, 2009
- Therapeutic Targets in the CMDs conference, July 9-11, 2009, Emory University, Atlanta. , Georgia Agenda
Resources
National Chemical Genomic Center Research Grants to support high throughput assay development and screens
RO3 HTS assay submission application (PAR-09-129). It rewards a total of $50K including the indirect cost. Three times per year are available for application. (http://grants.nih.gov/grants/guide/pa-files/PAR-09-129.html)
RO21 HTS assay development application (Par-08-024). It rewards a total of $100K for the direct cost. Two times per year are available for application. http://grants.nih.gov/grants/guide/pa-files/par-08-024.html
dystroglycanopathy_ models.doc
Wellstone Center, U of Iowa, Muscle Tissue and Cell Culture Repository
http://www.medicine.uiowa.edu/mdcrc/coreB.asp
Director: Steven Moore, MD, PhD
The Repository currently contains skeletal muscle biopsies from approximately 3000 neuromuscular disease patients and fibroblast cultures from approximately 10 muscular dystrophy patients. De-identified information about individual biopsies or cultures in the Repository can be found in a searchable database by clicking on the Database link on website above. Research scientists may request tissue from specific biopsies or cell cultures from specific patients by completing the appropriate forms. In order to accrue new biopsies or cell cultures in the Repository, patient consent forms must be completed.
Eurobiobank: CMD Cell Lines
http://www.eurobiobank.org/en/services/ebank/tissues.php
Eurobiobank is a cooperative project linking scientific resources for rare disease, CMD included, between 12 institutional biobanks. Eurobiobank resources are available to international scientists. After following the above link, type in “congenital” in the disease filter (not congenital muscular dystrophy). To access CMD cell resources, email the contact person for that particular biobank.
Cure CMD SMAB Members
Carsten Bonnemann, MD (Cure CMD SMAB Chairman)
Carsten Bonnemann
Markus Rüegg, PhD
Markus Rüegg
Dr. Markus Rüegg is Professor for Neurobiology at the Biozentrum, University of Basel, Switzerland. He studied Biochemistry and Neurobiology in Zurich and Stanford and was co-founder and member of the management of MyoContract, now called Santhera Pharmaceuticals, a biotech company that dedicated to develop therapies for neuromuscular diseases. Dr. Rüegg is an internationally recognized expert in neuromuscular research and has published numerous scientific papers in the field of neuromuscular research. In recent years, his work has also been devoted to the study of therapeutic interventions in mouse models for congenital muscular dystrophy. Dr. Rüegg is also partner of the network TREAT-NMD.
Volker Straub, MD
Volker Straub
Volker Straub, Professor of Neuromuscular Genetics, was trained as a Paediatric Neurologist at the University of Düsseldorf and the University of Essen in Germany. After his PhD thesis on Duchenne muscular dystrophy he worked as a postdoctoral research fellow in the laboratory of Kevin Campbell at the University of Iowa. In 2003 Volker joined the Institute of Human Genetics of Newcastle University, UK. Volker is joint co-ordinator of TREAT-NMD (www.treat-nmd.eu), executive board member of the World Muscle Society and executive board member of the Institute of Human Genetics at Newcastle University. Together with Hanns Lochmüller, Volker was responsible for setting up the German muscular dystrophy network, MD-NET, of which he was joint coordinator until 2008. Within the neuromuscular research group at Newcastle, Volker has a long-standing interest in the pathogenesis of muscular dystrophies, with research using zebrafish and mouse models. His current research also involves the application of contrast enhanced MRI.
John Porter, PhD
John Porter
Dr. John Porter is Program Director at the National Institute for Neurological Disorders and STroke (NINDS). He manages a portfolio of research grants that focuses on diseases affecting the motorneuron (spinal muscular atrophy), axon (inherited and acquired peripheral neuropathies), neuromuscular junction (inherited and acquired myasthenia gravis and slow channel syndrome) and skeletal muscle (myotonic dystrophy and congenital, Duchenne, Emery-Dreifuss, facioscapulohumeral, limb girdle, and oculopharyngeal muscular dystrophies). He also currently serves as the Excecutive Secretary for the interagency Musuclar Dystrophy coordinating Committee. Dr. Porter received his undergraduate degree in biology from the College of William and Mary and his PhD in Anatomy from Medical College of Virginia and completed his postdoctoral training in systems neurophysiology at the University of Alabama at Birmingham. Prior to joining the NIH, Dr. Porter was Professor of Neurology at Case Western Reserve University. His 20+ year academic research career focused on extraocular muscle biology in health and disease, including the mechanisms responsible for its novel responsese to a variety of neuromuscular disorders.
Alexandre Mejat, PhD
Alexandre Mejat
32 years old, Alexandre Mejat was first diagnosed with a muscular dystrophy when he was five and has been diagnosed with a Bethlem myopathy (Collagenopathy) at 24. He studied molecular and cellular biology in the Ecole Normale Superieure of Lyon (France) and obtained his PhD in biology after studying muscular atrophy mechanisms. Since 2005, he is a post-doctoral fellow at the National Institutes of Health (NIH, Bethesda, USA) in Dr Tom Misteli laboratory studying muscular laminopathies (Emery-Dreifuss Muscular Dystrophy). Since 2004, Alexandre is administrator of the Association for stem cells study (I-Stem institute, Evry, France, www.istem.eu) and an international expert for the scientific committee of the French Association against Myopathies (www.afm-france.org). He joined the Scientific and Medical Advisory Board of CureCMD in 2009.
Anne Rutkowski, MD
Anne Rutkowski
Anne Rutkowski, MD is a co-founder and Vice-Chairman of Cure CMD. Dr. Rutkowski is a practicing board certified emergency medicine physician in Los Angeles. Dr. Rutkowski’s daughter has congenital muscular dystrophy, subtype, dystroglycanopathy. Dr. Rutkowski graduated from the University of California Irvine Medical School, elected to Alpha Omega Alpha Honor Medical Society. She attended Bryn Mawr College as an undergraduate, graduating Magna cum laude with Honors in Biology. Prior to attending medical school, Dr. Rutkowski taught for 3 years in an inner city elementary school in Los Angeles, as part of Teach for America. She would like to shrink the diagnostic odyssey, see improvements and standardization of guidelines in medical care for all patients with CMD and a focused approach to identifying therapeutic targets. As a former educator, education and improved disease awareness are two further areas of focus for Cure CMD.
Edward Kaye, PhD
Edward Kaye
Edward M. Kaye, M.D. is currently Group V.P. and Therapeutic Area Head in Clinical Research at Genzyme Corporation where he supervises the clinical research in the lysosomal storage disease programs and in the genetic neurological disorders. He received his medical school education and pediatric training at Loyola University Stritch School of Medicine and University Hospital, Child Neurology training at the Boston City Hospital, Boston University, and completed his training as a Neurochemical Research Fellow (Geriatric Fellow) at the Bedford VA Hospital, Boston University in 1983. He was head of the section of Neurometabolism, Pediatric Neurology at The Floating Hospital for Children (Tufts University) and research fellow in gene therapy at the Massachusetts General Hospital until 1996 when he moved to Philadelphia to become Chief of Pediatric Neurology and Director of the Barnett Mitochondrial Laboratory at St. Christopher’s Hospital for Children. In 1998, he accepted the appointment as Chief of Biochemical Genetics at the Children’s Hospital of Philadelphia and Associate Professor of Neurology and Pediatrics until moving to Genzyme Corporation at the end of 2001. He continues as a member of the Neurology Department at the Children’s Hospital of Boston and has been on the editorial boards of a number of journals including Annals of Neurology, Journal of Child Neurology, and Pediatric Neurology. He is also on the Scientific and Medical Advisory Boards of the United Leukodystrophy Foundation, Spina Muscular Atrophy Foundation and Prize4Life.
Petra Kaufmann
Picture and Bio forthcoming.
Francesco Muntoni
Picture and Bio forthcoming.
