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A new clinical trial is meant to find treatments that can slow or stop the progression of motor neurone disease (MND). This disease causes progressive weakness and is fatal, making the need for an effective treatment high. This trial, which is called MND-SMART, is being conducted across the United Kingdom. It will include as many people with MND as possible, regardless of the stage or treatments they have already received. Researchers hope that this new trial, which tests many drugs rather than just one, will be more successful in finding a treatment for MND.
About Motor Neurone Disease (MND)
MND is a rare, progressive disease that causes weakness. It affects the brain and nerves, which then leads to weakness in muscles across the body. It mainly affects those who are in their 60’s and 70’s.
Symptoms include weakness in the legs or ankles, which can cause trips and make it difficult to climb stairs, slurred speech, difficulty swallowing food, weak grips, which can make it hard to carry or open things, muscle cramps and twitches, weight loss, and an inability to stop oneself from laughing or crying.
This disease occurs when the motor neurons in the brain slowly stop working. It is not known why they deteriorate. In most cases it is not hereditary, but one may be at a heightened risk if a family member has it. There is a higher likelihood of developing it if one has frontotemporal dementia as well.
A diagnosis may be hard to obtain in the early stages, as symptoms are not obvious. A diagnosis usually comes after ruling out other conditions. Tests to accomplish this may include blood tests, scans of the brain and spine, measurements of the electrical activity in the muscles and nerves, and a lumbar puncture.
After one has a diagnosis, treatment is symptomatic, as there is no cure. Occupational therapy can help with everyday tasks, while physical therapy can help to maintain muscle strength. Speech therapy may also be helpful. A specific diet may be recommended, as will emotional support. A drug called riluzole may be prescribed, as it has been shown to slightly slow the progression. Medications to stop muscle stiffness and salivary problems are other options for treatment.
Hundreds of people have enrolled in this study, which differs from conventional drug trials. More than one medication will be tested at one time, as there will be a shared placebo group and multiple groups who receive a medication. This not only allows more than one drug to be tested, but it allows for more patients to receive treatment.
Not only are there hundreds of participants in this study, but it is designed to be adaptive. This means that researchers will be able to change the study as they receive the results. The first step in this massive study is to test drugs that have already been approved in order to see if they have any effect on MND.
This study is expected to begin in Edinburgh, with other clinics across the U.K. following as 2020 progresses. If one is living with MND, they can apply for this study at http://www.MND-SMART.org.
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