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New drug shows hope for infants with muscle disease
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A new experimental drug may have the potential to improve the quality of life for infants suffering from a rare, lethal neuromuscular disorder, US researchers have found.Spinal muscular atrophy is a genetic disease that affects around one in every 11,000 births.It affects the nerve cells in the spinal cord that connect to the muscles, causing them to waste away resulting in progressive muscle weakness and difficulty in breathing and eating.Infantile-onset, which is the most severe form of the disease, occurs from a defect in the gene called SMN2, responsible for producing survival motor neuron (SMN) — a protein critical for normal cell function.
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Novel drug rescues babies with fatal neurodegenerative disease
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There have been few happy endings when it comes to spinal muscular atrophy (SMA), the most common genetic cause of death in childhood.The disease inexorably destroys the motor neurons of the spinal cord and brainstem that control movement, including swallowing and breathing.In its most severe form, SMA kills those afflicted at about age 2, most commonly by suffocating them.There are no Food and addiction Administration (FDA)–approved drugs for the disease.
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Alzheimer's drug may have mild effect on disease
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The Alzheimer's drug solanezumab has some effect on the disease after all, a top expert on the disease said at an international conference held in San Diego.Solanezumab failed to demonstrate statistically significant evidence of clinical efficacy according to recent results from a trial, known as Expedition 3.That trial primarily measured solanezumab on its ability to slow cognitive decline in patients with mild Alzheimer's disease.A Thursday evening panel at the Clinical Trials on Alzheimer's Disease conference discussed secondary results released at the conference.
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New drug might halt the progress of neurodegenerative disease
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Spinal muscular atrophy type 1 is a hereditary disease that causes severe muscle weakness in infants and children.Until now, there was no known cure or disease-modifying treatment for the condition.Clinical trials of a new drug called nusinersen, however, show significant promise.A new drug has shown positive effects in infants diagnosed with SMA-1.
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