Duchenne Muscular Dystrophy

Duchenne muscular dystrophy, also known as DMD, is a genetic disorder characterized by progressive muscle weakness and degeneration. Duchenne muscular dystrophy was first described in the 1860s by Guillaume Benjamin Amand Duchenne, a French neurologist but until the 1980s, little was known about the cause of any kind of muscular dystrophy. This disease is x-linked recessive.
Approximately two-thirds of DMD occurrences are hereditary while one-third of cases are due to a new mutation. It is one of nine types of muscular dystrophy. DMD is triggered by the absence of dystrophin, a protein which helps maintain muscle cells intact. This disease primarily affects boys with symptoms appearing between ages 3-5, but in rare cases, it can mildly affect …show more content…
According to a study published online in The Lancet, the above conclusions are in line with the typical prescription procedures in numerous health centers and are likely to influence parents who are hesitant about these therapies and whether or not their children should receive them.
DMD, which typically affects men due to the location of its causative genetic mutation, is characterized by loss of muscle function which begins in the lower limbs. As this devastating neuromuscular disease progresses, patients at some point often receive glucocorticoids. Studies since the late 1980s indicate short-term benefits to treating with these drugs, which include delaying the loss of strength in muscles as well as …show more content…
For those who received treatment for over 1 year, glucocorticoids hindered immobility milestones which affected lower limbs by 2.1 – 4.4 years, e.g., lying to standing, climbing four stairs, and walking/running 10 meters, equated to males that received the medication for less than a year.
Long-term therapy further hindered immobility milestones in upper limbs such as hand function, raising the hands to the mouth and performing a full overhead reach. Decreased death risk over the length of the study was also attributed to long-term use of these drugs. What's more, deflazacort, a glucocorticoid newly approved specially for DMD by the Food and Drug Administration, delayed loss of the ability to move from supine position to standing, walking and hand to mouth function notably better than prednisone, the most common glucocorticoid prescribed in the United States for DMD.
Dressman notes that glucocorticoids are currently a standard part of care for most DMD patients, with various clinics prescribing them as soon as a diagnosis is made. Future studies, he says, will examine which medicines in this class of drugs and which regimens are bound to offer the most benefits as well as how these benefits differ with long-term use of

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