A report on drug development in neuromuscular diseases as of January 2009
posted on January 1, 2009 - 2:37pm
In the era of molecular biology, the drug development process has moved from a “let’s try it and see what happens” approach to a scientifically based process of discovery and application.
For many of the diseases in MDA’s program, drug discovery begins with gene discovery — identifying a gene that, when flawed, causes a disease.
Thirty-eight new grants support research in ALS, central core disease, spinal muscular atrophy, muscular dystrophy, and other diseases in MDA's program
posted on August 17, 2010 - 6:39pm
MDA has awarded 38 new research grants totaling more than $14 million and covering more than a dozen neuromuscular diseases.
MDA's Board of Directors met in Los Angeles July 16, where it reviewed and approved the new grants based on recommendations from the MDA Scientific and Medical Advisory Committees. Grants were scored and recommended for approval based on the capabilities of the...
IDMC-8, held Nov. 30-Dec. 3, 2011, explored disease mechanisms, clinical aspects and ideas for future therapies in myotonic muscular dystrophy
posted on December 8, 2011 - 3:05pm
The 8th International Myotonic Dystrophy Consortium Meeting (IDMC-8), was an exciting mix of the latest scientific developments and clinical research in types 1 and 2 myotonic dystrophy (MMD1 and MMD1, also known as DM1 and DM2).
University of Illinois-Chicago scientist receives $530,480 to test immune-system modulator for myasthenia gravis
posted on March 14, 2011 - 3:51pm
MDA’s translational research program has announced it is funding research into a potential treatment for the autoimmune disorder myasthenia gravis (MG).
The grant of $530,480 to University of Illinois-Chicago professor Matthew Meriggioli will fund a three-year study of an immune-system modulator called GM-CSF. The compound will be tested in a mouse model of MG and in a small group of human...
Repligen Corp. is proceeding with development of an experimental compound to treat Friedreich's ataxia, with help from a new MDA grant.
posted on December 17, 2009 - 1:47pm
Development of a promising experimental medication to treat Friedreich's ataxia (FA) is proceeding, with help from a $731,534 grant MDA awarded to Repligen Corp. of Waltham, Mass., this month (December 2009).
This is the second research grant that MDA has awarded to the small biopharmaceutical company, through the Association's translational (laboratory-to-clinic) research program.
A new, NIH-supported translational research center will focus on exon skipping as a treatment for MD
posted on October 19, 2011 - 5:00am
A Center for Research Translation of Systemic Exon Skipping in Muscular Dystrophy has been established by the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), part of the U.S. National Institutes of Health.
MDA's research program in 2010
posted on December 31, 2009 - 1:10am
As MDA's research program heads into 2010, new directions, strategies and partnerships are under way.
All have the ultimate goal of moving research through the “pipeline” from basic science discoveries in university laboratories, to biotechnology development, and ultimately into viable therapies that can be prescribed in the clinic.
Below are examples of how this “pipeline” research approach is...
MDA Venture Philanthropy—not your father's research program
posted on April 1, 2009 - 3:45pm
An increasingly popular trend in funding research projects is “venture philanthropy,” which applies effective business principles and practices to nonprofit goals.
Now, MDA has put this model to work for its research program by creating a new nonprofit spin-off, MDA Venture Philanthropy (MVP), that seeks to overcome barriers and speed the development of drugs for the diseases in MDA’s program.
'Muscle Gene Therapy' brings together the latest research in this promising field
posted on March 8, 2010 - 11:19am
MDA grantee Dongsheng Duan has published a book on the latest advances in gene therapy for muscle disease, particularly muscular dystrophies.
Duan, a professor of microbiology and immunology at the University of Missouri, served as editor of the book Muscle Gene Therapy and co-authored two of its 15 chapters. Duan is currently researching gene transfer therapy in dogs.
About the new book
An MDA-sponsored meeting explored progress in five key therapeutic strategies under development for neuromuscular diseases
posted on March 18, 2011 - 3:30pm
Moving therapeutic strategies from the laboratory to clinical trials and ultimately to the market as treatments was the theme of the MDA National Scientific Conference held March 13-16, 2011, in Las Vegas.
Some 300 people attended the conference, the first in a planned series of such MDA-sponsored meetings that will emphasize new research and current medical care. The majority of presenters and...
