Tatiana Schlossberg, the youngest granddaughter of President John F. Kennedy, recently revealed she was diagnosed with ...
Indian researchers identify a novel USP18 gene mutation linked to pseudo-TORCH syndrome, aiding treatment for affected ...
Rutgers neuroscientist Peng Jiang and his neuroscience colleague Mengmeng Jin have made a discovery they say could reshape ...
In many aspects of our lives, we find meaning in the order in which events occur. We buy into myths about "middle child ...
A new gene-editing technique called PERT offers a potential single-tool solution for numerous genetic disorders caused by ...
Novartis secures FDA approval for Itvisma, a one-time gene therapy designed to boost motor function across a broad SMA ...
Technical advances allow researchers to trace the genetic changes that occur over time.
Instead of requiring personalized gene edits for each patient, the new approach could create a standardized method to use for ...
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FDA Gives Broad Approval to Gene Therapy for Rare Neurologic Disease
The FDA approved the intrathecal gene therapy onasemnogene abeparvovec (Itvisma) to treat spinal muscular atrophy (SMA) in ...
Indian researchers have identified a novel gene mutation causing pseudo-TORCH Syndrome Type 2 in a child from a consanguineous marriage, using exome sequencing.
A new study by researchers at the University of Wisconsin–Madison provides the first empirical evidence connecting the ...
Itvisma contains the active ingredient in Zolgensma approved in 2019 for children younger than 2 years but is reformulated for intrathecal administration across age groups.
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