The start of any new gene therapy trial is an exciting and nervous time. But for the participants and doctors involved in the recently launched trial for Duchenne Muscular Dystrophy (DMD) at Columbus ...
ITVISMA is a prescription gene therapy used to treat adults and children 2 years of age and older with spinal muscular atrophy (SMA). ITVISMA is given as a one-time intrathecal injection. What is the ...
Scientists have discovered that injecting a novel human protein into muscle affected by Duchenne muscular dystrophy significantly increases its size and strength, findings that could lead to a therapy ...
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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 ...
The future of weight loss could look like this. Inject your muscles with a compound that helps them burn more calories than usual and then do your daily chores to shed those extra pounds. That's the ...
Peptides, short chains of amino acids, play key roles in the body. FDA-approved drugs like insulin and GLP-1 medications are ...
Itvisma, an intrathecal version of Zolgensma, can be used in children, teens and adults to stabilize or improve motor ...
Injections for spinal muscular atrophy (SMA) refer to two treatments that the Food and Drug Administration (FDA) has approved for SMA: nusinersen (Spinraza) and onasemnogene abeparovec-xioi (Zolgensma ...
Injecting a novel human protein into muscle affected by Duchenne muscular dystrophy significantly increases its size and and strength, discovers scientists in new findings. These findings could lead ...
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