Many patients with myotonic dystrophy feel tired. Clicians see these as fatigue which can affect a patients life significantly. A recent study that we are reviewing stated that it may be a combination of fatigue and depression. Depression has been noted in many myotonic dystrophy patients. Here is the conclusion of the study
In summary, these data shows a significantly higher prevalence of perceived fatigue in patients with DM1 than in matched healthy control subjects, with an experienced impact on physical and psychosocial domains.These findings support the inclusion of fatigue as a main target for treatment interventions aimed to reduce fatigue through cognitive behavioral therapy and graded exercise. Such an intervention was recently applied and shown to reduce fatigue,increase activity and improve social participation [38].
This month a new company Dyne Technologies was formed pioneering targeted therapies for muscle diseases, with an initial focus on Myotonic Dystrophy. This Company has been funded with $50 million in a series A round which is a solid foundation to begin a process to identify a treatment. The approach they are using focuses on muscle. Of course, myotonic dystrophy is more than just muscle weakness so other organs and systems may be helped as well.
We are advancing treatments for serious muscle diseases, with an initial clinical focus on myotonic dystrophy type 1 (DM1), a rare, inherited disorder that causes muscle-wasting as well as cardiac and pulmonary dysfunction.
For each of the estimated 40,000 people in the U.S. living with DM1, the underlying cause of the disease is a genetic mutation in the DMPK gene. Despite clear drug targets and well-understood biology, drug development efforts have not resulted in effective therapies, largely due to the inability to deliver enough medicine to muscles while minimizing exposure to other tissues. Through our revolutionary FORCE platform, we aim to bring the first disease-modifying treatments to patients and families impacted by DM1.
How does it work????
We have designed a therapeutic molecule by linking an antibody to an oligonucleotide.
The receptor is highly expressed on muscle cells. By engaging the receptor, the antibody brings the oligonucleotide to the primary site of disease and facilitates delivery into the muscle cells.
Once inside the cell, the oligonucleotide binds with the RNA that is driving disease progression.
Disease-causing RNA is degraded.
This highly targeted approach is designed to promote muscle health.
A new review article has been published. An exercise or training program is generally good for people with Myotonic Dystrophy. However, not enough research has been done to figure out the optimum workout program.The article is a bit technical but its good as a general review. Its also good to print out and take to your doctor.
Bladder and urinary issues in Myotonic Dystrophy are not reported as well as they should be. Many patients report incontinence problems but this is not listed on many of the myotonic dystrophy sites as an issue. During hospitalization incontinent issues may be of greater visualization as hospitals now have bladder scanners to look at this issue. This article based on a small sample size shows there is a variety of urinary bladder issues of various nature. More work is needed to see how this issues can be overcome.
The issues with incontinence are many as shown in the attached study. Retention issues arise as the hospitals use 400ML of urine before they want to catheterize.
Recent information came to light that Ionis is still continuing pursue a drug for myotonic dystrophy. The first trial ended and was not successful. What we learned is that the team was impressed that progress was made. Rather than pursue this initial drug they may be switching to a new more highly improved drug. This makes sense as the cost to pursue a drug is high $$$$ and you want your best candidate forward.
