Other
Novartis to buy US gene therapy group AveXis for $8.7 bn
Swiss pharmaceuticals giant Novartis announced Monday it will buy US gene therapy company AveXis for $8.7 billion (7.1 billion euros).
Apr 9, 2018
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Page 16 - News tagged with muscular atrophy
Pediatrics
The dying child: Room for improvement in end-of-life care
Many pediatricians and pediatric subspecialists believe that their clinical care extends from treating ill children through end-of-life care. However, are pediatricians actually meeting the needs of families and their dying ...
May 27, 2016
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Medications
Sky-high drug prices for rare diseases show why Orphan Drug Act needs reform
When Marathon Pharmaceuticals announced in February it would market a drug for treating Duchenne muscular dystrophy for US$89,000 a year, the negative reaction was so intense that the company immediately suspended the rollout. ...
Mar 17, 2017
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Neuroscience
New drug improves motor function of children with genetic disorder
Children with later-onset spinal muscular atrophy (SMA) were more likely to show gains in motor function when treated with a new medication compared to children receiving a sham procedure, according to a study published today ...
Feb 15, 2018
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Medications
Lifesaving drug for infants costs $2.1 million a dose
(HealthDay)—Its extremely high price tag means that a lifesaving medication to treat young children with spinal muscular atrophy is simply too expensive for most families.
Jul 30, 2019
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Pediatrics
New drug enables infants with genetic disorder to live longer, gain motor function
Infants with the most severe form of spinal muscular atrophy (SMA) were more likely to show gains in motor function and were 47 percent more likely to survive without permanent assisted ventilation support when treated with ...
Nov 1, 2017
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Neuroscience
A pathogenic mechanism in motoneuron disease
Motor neurons are the nerves that send impulses to the muscles to generate movement. Damage of these neurons can cause very diverse diseases, for example spinal muscular atrophy in children or adult amyotrophic lateral sclerosis.
Nov 2, 2017
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Medical research
Researcher develops, proves effectiveness of new drug for spinal muscular atrophy
According to recent studies, approximately one out of every 40 individuals in the United States is a carrier of the gene responsible for spinal muscular atrophy (SMA), a neurodegenerative disease that causes muscles to weaken ...
Sep 15, 2014
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Medical research
Gene replacement therapy offers viable treatment option for fatal disease
Spinal muscular atrophy (SMA) is a disease that causes progressive degeneration in the nerve cells that control muscles, thereby causing muscle weakness and eventually death. SMA affects approximately 200,000 people in the ...
May 5, 2016
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Parkinson's & Movement disorders
New neuromuscular model promises to revolutionize high-throughput drug screening studies
In neuromuscular diseases, neurons and muscle cells stop communicating properly. Researchers led by Mina Gouti can now model this in 2D in a culture dish. Writing about their findings in Nature Communications, they say the ...
Dec 19, 2023
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