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New drugs are helping children with a fatal muscle disease walk again
 

New drugs are helping children with a fatal muscle disease walk again

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Spinal muscular atrophy (SMA) is a very rare genetic disorder that often leads to early death in young patients, but a new experimental drug has produced some incredible results in a recent trial - it's even enabled several children with the...
 
www.sciencealert.com
 
7 Oct 2016 - General
 
Muscular dystrophies are very rare but once you have it, that is bad. As I am working in the largest childrens hospital in the world I have some experiences with the patients with muscular dystrophies. They are transmitted from a generation to the next generation since they are genetic disorders. Duchenne is the worst one to have because once a child is having the disease it is 100% fatal and usually they live 15- 18 years. It occurs once in 2400 births. Becker muscular dystrophy is less severe ...
 (Total 146 words)
Nusinersen is giving us a glimmer of hope in providing treatment for kids who have been diagnosed with spinal muscle atrophy. There is not much more in our medical careers that could break our hearts than seeing helpless kids struggle and experience delay with motor development. I am crossing my fingers that this medicine will pass safety issues and become available soon to treat all the three types of SMA. Let this be the gift for persevering parents of brave SMA patients as they course through...
 (Total 114 words)
Though still under clinical trials, Nusinersen is being considered as a hope for treatment of spinal muscle atrophy. It has already proven its efficacy to improve muscle strength significantly. Many children who are unable to walk, may get a chance to stand up and go forward. It is not still clear in what extent the disease will be cured.Complete cure of a genetic disorder may not be possible. But if the children can lead a better life, it will be incredible. An application for FDA approval is s...
 (Total 99 words)
I am well aware of Spinal Muscular Atrophy (SMA) as two kids of my cousin are suffering from this disease. It is a genetic disorder that first affects the limbs and restricts the movement than it results in an early death of young patients. A recent research has shown that a drug Nusinersen is incredible in treating SMA. It has been administered to control group of children that shows positive results. Children who have been unable to even crawl since many years are regaining strength gradually....
 (Total 134 words)
An experimental drug, that is still in its early stages of clinical trials have shown promising results in patients with spinal muscular atrophy. Spinal muscular atrophy is a rare neuromuscular disorder characterized by loss of motor neurons and progressive muscle wasting, often leading to early death in children. The new drug have been so successful that it has been able to make some children who suffer from this disease to walk again. Something they never would have thought would be possible. ...
 (Total 110 words)