Medical research
Scientists show 'matchmaker' role for protein behind SMA
A puzzling question has lurked behind SMA (spinal muscular atrophy), the leading genetic cause of death in infants.
Feb 14, 2017
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Spinal Muscular Atrophy
Medical research
Experiments in mice may help boost newly FDA-approved therapy for spinal muscular atrophy
Johns Hopkins researchers along with academic and drug industry investigators say they have identified a new biological target for treating spinal muscular atrophy. They report they have evidence that an experimental medicine ...
Jan 9, 2017
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Medications
FDA OKs first treatment for rare genetic disorder
The Food and Drug Administration on Friday approved the first treatment for children and adults with spinal muscular atrophy, a rare genetic disorder marked by progressive muscle weakness that's the most common genetic cause ...
Dec 23, 2016
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Medical research
Mechanisms underlying direct programming of stem cells into motor neurons could lead to cell-replacement therapies
A team of scientists has uncovered details of the cellular mechanisms that control the direct programming of stem cells into motor neurons. The scientists analyzed changes that occur in the cells over the course of the reprogramming ...
Dec 8, 2016
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Medical research
Stanford patient is first infant to receive lifesaving drug for neurodegenerative disease
In 2013, Zoe Harting became the first baby in the world to receive an experimental drug that her doctors hoped would save the lives of thousands of infants like her.
Dec 6, 2016
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Medical research
Study finds new treatment for spinal muscular atrophy safe for infants
Infants as young as five weeks old with the most severe form of spinal muscular atrophy (SMA) - a leading genetic cause of infant mortality - can be treated safely with nusinersen. This investigational treatment slowed progression ...
Dec 6, 2016
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Medical research
Trial offers hope of a treatment for spinal muscular atrophy
A research team led by the University of Oxford has found a promising treatment for degenerative disease spinal muscular atrophy (SMA), a leading genetic cause of child death.
Sep 20, 2016
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Genetics
Potential new target for treatment of spinal muscular atrophy discovered
For the first time, scientists found that in spinal muscular atrophy (SMA), the affected nerve cells that control muscle movement, or motor neurons, have defects in their mitochondria, which generate energy used by the cell. ...
Sep 15, 2016
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Medical research
Scientists locate possible therapy target for spinal muscular atrophy
Researchers at Iowa State University have discovered what could lead to a new treatment for spinal muscular atrophy, a potentially fatal genetic disorder in young children.
Aug 9, 2016
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Medical research
Candidate drug for spinal muscular atrophy, with roots in CSHL research, passes major hurdle
In an announcement that delighted the families of patients and surprised many pharmaceutical industry analysts, the biotechnology firms Biogen and Ionis Pharmaceuticals on Monday said they had decided to cut short a phase-3 ...
Aug 4, 2016
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