Some 1 million people around the world suffer severe neurological problems, such as epilepsy, motor impairment and cognitive dysfunction, because they express insufficient SynGAP1, a GTPase-activating ...
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REGN Reports Encouraging Data on Gene Therapy for Genetic Hearing Loss (Revised)
Regeneron Pharmaceuticals, Inc. REGN announced updated data on its investigational gene therapy DB-OTO from the CHORD study.
This article outlines common CMC problems that are obstacles that steal momentum and create costly detours in the development ...
Using fruit flies, University of Wisconsin–Madison researchers have developed a new model for investigating the genetic ...
In a study published in Nature Medicine, Leone and her team reported the first targeted gene therapy to restore myelin, the ...
Despite announcing a broad pivot to siRNA earlier this year, Sarepta is following through with an investigational gene ...
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