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.
John Crowley talks about what's needed in the "golden age" of drug development
posted on December 31, 2009 - 1:19am
John Crowley is the father of two children with Pompe disease (acid maltase deficiency). His quest to develop a drug to save their lives is chronicled in the movie "Extraordinary Measures," to be released by CBS Films in January 2010.
Crowley was interviewed for the Quest article "Heroes, Hope & Hollywood." Below are more of his thoughts on the "golden age" of drug development and how...
posted on January 1, 2011 - 4:42pm
A biomarker is any biological indicator that doctors or researchers can objectively measure and evaluate to determine the state of an individual’s health; confirm disease onset and progression; or gauge whether an experimental treatment is working or not.
In this first of several reports on MDA's Clinical Conference, the role of genetics and immunology in different neuromuscular diseases is described
posted on March 19, 2012 - 3:08pm
More than 500 physicians, allied health care professionals and MDA staff attended the MDA's 2012 Clinical Conference in Las Vegas, March 4-7.
The program emphasized:
Progress is ongoing in coaxing stem cells along specific paths, altering their genes and understanding the immune response to stem cell transplantation
posted on June 13, 2011 - 2:23pm
Stem cells are a hot topic these days in medicine, science and law, although the term has multiple meanings and it's easy to get confused.
In short, stem cells are cells at an early stage of development from which specialized cells, such as muscle or nerve cells, can develop (in other words, from which these specialized cells "stem").
Different kinds of stem cells are referred to as:
An update on stem cell research related to neuromuscular disease as of July 2009
posted on July 1, 2009 - 5:17pm
Stem cells — immature cells with the potential to develop into different tissue types — have been heralded as a major advance for developing treatments for a variety of diseases. That’s true for diseases of the nerves and muscles, where such cells could potentially be transplanted into the body and either support or replace a patient’s ailing cells.
Studying the mechanisms controlling gene activity
posted on December 31, 2009 - 1:15am
Slip the word “genetics” into a casual conversation and you likely won’t get too many questioning looks.
Most people basically understand that “genetics” refers to DNA and genes, the blueprint of life, the code that determines whether someone has blue eyes or green. Many are familiar, too, with the idea that “genetics” can cause a variety of health conditions: diabetes, breast cancer, muscular...
Two federal agencies, the NIH and FDA, are cooperating in an attempt to reduce the time it takes to approve new drugs.
posted on February 26, 2010 - 4:45pm
The National Institutes of Health (NIH) and the U.S. Food and Drug Administration (FDA) have announced plans to establish a Joint NIH-FDA Leadership Council, with the goal of shortening the time it takes to move promising therapies through the regulatory process and into the hands of waiting patients.
Video games can serve the higher public good when players work alone or with a team to solve genetic and biochemical puzzles
posted on March 31, 2011 - 11:57am
Note: This article was updated Sept. 19, 2011.
Video games have a reputation for being big time-wasters. But what if you could help solve scientific mysteries — such as unraveling the origins of a genetic disease — at the same time as you’re having fun?
That’s the idea behind several new online games designed to harness the collective brain power of computer users — a process known as “...
ALS TDI's first-quarter 2010 research update can be summed up with one word: momentum.
posted on January 27, 2010 - 4:02pm
Significant progress on multiple fronts was reported by ALS TDI (the Amyotrophic Lateral Sclerosis Therapy Development Institute) in its first Webcast of 2010, hosted on Jan. 14.
