2024 is already off to a busy start for Mon4t!

Over the last two weeks, patients from across the globe have contacted Mon4t in search of solutions for various rare genetic conditions including Amyotrophic Lateral Sclerosis (ALS), Myasthenia Gravis (MG) and Wolfram Syndrome. Each of these conditions is manifested by several symptoms and characterized by progressive neurodegeneration.

ALS impairs the motor nerve cells in the spinal cord and brain affecting limb control and breathing. The degeneration of upper and lower motor neurons causes muscle weakness, spasms and atrophy hindering daily activities as the condition progresses. MG is a chronic autoimmune disorder that causes skeletal muscle weakness of the eyes, mouth, throat and limbs due to breakdown in communication between nerves and muscle. Wolfram Syndrome is a rare autosomal recessive disorder affecting the central nervous system, particularly the brainstem. These diseases can also manifest psychiatric symptoms, requiring a holistic approach to treatment.

For each of these conditions there is no direct treatment, therefore patients are desperately exploring their options. As these conditions are all rare, there is less incentive for the medical industry to develop new treatments. When clinical trials do take place, it is hard to recruit subjects insufficient numbers. Even if sufficient subjects are found very few are assessed at every site. Therefore, inter-rater variability increases and obtaining statistical significance is more challenging compared to other conditions.

Another setback is that there are very few experts in every country (if at all) who specialize in treating these conditions. Thus, for the limited treatment that may be available, geographical distance is a significant obstacle for these patients, and reaching the expert for evaluation is challenging, if at all possible.

Mon4t bridges the gap between the patients and the PI in clinical trials, as well as between them and their physicians. These patients found Mon4t and we are now engaged with their patient organizations for these conditions, as well as pharmaceutical companies who develop drugs to address them. Together we work to become a part of the patient journey, assisting them in every step of the way and also raise awareness so that additional research and solutions can be established.