The behavioral findings demonstrate similarities
in progressive muscle weakness and a decline in motor coordination in both models of motor neuron disease with a later onset and slower progression noted with PFN1 animals.
Using CRISPR / Cas9, the Hamon Center team was able to correct the genetic defect in a mouse model of DMD and thus prevent the development of features of the disease, which in boys
causes progressive muscle weakness and degeneration, often along with breathing and heart complications.
The findings demonstrate similarities
in progressive muscle weakness and a decline in motor coordination in both models of motor neuron disease with a later onset and slower progression noted with PFN1 animals.
It is caused by genetic mutations that result in the loss of the dystrophin protein, leading to
progressive muscle weakness and death by the second or third decade of life.
Patients with peripheral neuropathies may experience pain, tingling, numbness and
progressive muscle weakness.
Spinal muscular atrophy is a genetic disease that causes the degeneration of spinal cord motor neurons and leads to
progressive muscle weakness, atrophy and inability to walk or sit, and breathing difficulties.
Amyotrophic lateral sclerosis (ALS) is a motor neuron disease, which leads to
progressive muscle weakness and ultimately to lethal paralysis, while frontotemporal dementia (FTD), the second most common form of dementia (after Alzheimer's) in those under 65.
Progressive muscle weakness in the limbs may also be evident.
According to reports, the ECHR states that it would be kinder to let the child die, and that keeping the baby on life support would prolong his suffering given that he can not recover from his disease, which causes
progressive muscle weakness, including in key organs such as the heart.