The advent of therapeutic interventions for spinal muscular atrophy (SMA) has increased the importance of presymptomatic diagnosis and treatment. When to start treatment in children with less severe disease remains controversial. ...
Jun 11, 2020
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The enzyme Gemin3 was identified as the molecular 'bridge' between genes whose mutation or disruption causes amyotrophic lateral sclerosis (ALS), according to a new study in Nature's Scientific Reports from scientists at ...
Jan 16, 2020
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Four-month-old Ainsley Cardente smiles, enjoys "helicopter" rides from her mom and protests when lunch is late. Perfectly ordinary, all of it. And her life is expected to remain that way thanks to a $2.1 million drug recently ...
Jan 6, 2020
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Swiss pharmaceutical company Novartis is preparing to give away 100 doses of the world's most expensive drug, which treats a rare childhood disorder, but its recipient selection process has drawn criticism.
Dec 20, 2019
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Neuroscientists at Lund University in Sweden have developed a new technology that engineers the shell of a virus to deliver gene therapy to the exact cell type in the body that needs to be treated. The researchers believe ...
Dec 13, 2019
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Fresh insights into how nerves connect with muscles in the body could aid the development of therapies to treat neurodegenerative diseases.
Oct 30, 2019
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A method to create a faster and lower cost alternative for a gene therapy tool has been developed by Boston University School of Medicine (BUSM) researchers.
Sep 19, 2019
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(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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The University of Rochester Medical Center (URMC) has been tapped as one of the first institutions in the U.S. to offer a new gene replacement therapy to treat spinal muscular atrophy (SMA). The new treatment can be delivered ...
Jun 6, 2019
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Gene therapy—for so long something that belonged to the future—has just hit the streets.
Jun 5, 2019
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