Though often used to describe a specific condition, muscular dystrophy is actually a group of about 40 different muscle conditions.
All of these conditions share a number of common traits: progressive skeletal muscle weakness, death of muscle cells, limited range of motion. Also, all forms of muscular dystrophy (or MD) are mostly hereditary.
However, despite the strong similarities, their select differences often make it difficult to find effective treatments, let alone a potential cure.
But as research continues to advance, breakthroughs continue to happen. The most recent example involves a potential treatment for the most common form of MD:
Slowing the Progression
As mentioned before, MD is a progressive condition meaning signs and symptoms get worse over time as the muscles of the body get continually weaker. One of the initial goals of any treatment is to simply slow down that progression.
And a doctor named Mani S. Mahadevan has helped identify an experimental drug that might be able to do just that. Dr. Mahadevan actually has extensive experience working with MD. In fact, he was part of the team that discovered the gene responsible for myotonic dystrophy.
The drug in question isn’t actually new. In fact, it’s been tested and used in clinical trials for other condition such as rheumatoid arthritis. In regards to MD, it’s been tested on mice which resulted in improved moment and grip. Dr. Mahadevan believes it could have similar positive results in humans.
Since it’s already been used in clinical trials for other conditions, the process of getting it tested for MD and available to consumers could happen much faster than it would for new drugs.
Though Dr. Mahadevan isn’t sure when clinical trials might begin, he’s very optimistic about the future. “There’s a log of hope” he says.
Once tested and proven safe for those with MD, the drug can be reviewed by the FDA and made available to patients.
About Myotonic Dystrophy
Myotonic dystrophy is a form of muscular dystrophy characterized by the slow, progressive wasting of the muscles as well as cataracts, heart conduction defects, and myotonia. Passed hereditarily, myotonic dystrophy affects men and women equally and can set in during any age.
Myotonic dystrophy is caused by an expanding piece of DNA that grows worse with each generation. It’s likely that one of the parents of someone with myotonic dystrophy had the DNA defect as well, just less severe. Therefore, it wouldn’t have produced any symptoms in the parent.
Myotonic dystrophy is split predominately into two main groups: DM1 and DM2. DM 1 is generally more severe than DM2 though the severity in both cases can vary considerably. Though both forms can have a significant impact on a person’s life, death is unlikely in either case.
Rollx Vans and MDA
To help raise awareness for muscular dystrophy, Rollx vans, a lead distributor of wheelchair vans nationwide, partners with organizations such as MDA. For every van we sell, customers can choose an organization like MDA for us to donate to.
To learn more about muscular dystrophy, make sure to visit the MDA website.
Could this new drug impact you or someone you know? What is your experience with myotonic dystrophy?